BMRN

Detailed Phase 3 CANOPY-HCH-3 data in children living with hypochondroplasia also featured in a late-breaking oral presentation at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting VOXZOGO demonstrated statistically significant improvements across multiple measures of growth, including annualized growth velocity, standing height, height Z-score and arm span BioMarin recently submitted a supplemental New Drug Application (sNDA) to the FDA to support expanding treatment with VOXZOGO to include children with hypochondroplasia SAN RAFAEL, Calif., Sept. 9, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data from the Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO® (vosoritide) in children with hypochondroplasia were published in New England Journal of Medicine (NEJM) Evidence and presented at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting. The data included new results on the magnitude of benefit seen in children receiving VOXZOGO, including statistically significant improvements in annualized growth velocity (AGV), standing height, height Z-score and arm span after 52 weeks, with safety findings consistent with the established profile of VOXZOGO in achondroplasia. The CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint (least squares [LS] mean difference of 2.33 cm/year; p<0.0001). Children treated with VOXZOGO also showed significant improvements in standing height (LS mean difference of 2.35 cm; p<0.0001), height Z-score (LS mean difference of 0.39 standard deviation score; p<0.0001), and arm span (LS mean difference of 1.03 cm; p=0.0082) compared with placebo. Children who received VOXZOGO also demonstrated numerical improvements in quality of life, and follow-up will continue to assess the impact of treatment over a longer term. The overall safety profile was consistent with previous studies of VOXZOGO, with most adverse events reported as mild and no treatment-related serious adverse events identified. "These results presented in detail for the first time provide a comprehensive picture of the impact of VOXZOGO across multiple measures of growth in children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Based on this compelling body of evidence, we have submitted these data to the FDA with the goal of securing approval for the first medicine for children with hypochondroplasia." "Hypochondroplasia can affect a child's growth, physical function and everyday life, with families often navigating unique challenges as they support their children's development," said Andrew Dauber, M.D., lead study investigator and Chief of Endocrinology at Children's National in Washington, D.C. "The changes we observed in annualized growth velocity and arm span provide encouraging evidence of how children with hypochondroplasia responded to treatment throughout the study. These findings deepen our understanding of the condition while reinforcing VOXZOGO's potential as the first targeted medicine developed specifically for children with hypochondroplasia." BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia and are on track with the submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch. Below are key BioMarin presentations across both achondroplasia and hypochondroplasia at ESPE, with all times listed in Central European Summer Time: Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial ResultsOral Presentation #LBA 1067Wednesday, Sept. 9, 10:48 – 10:56 a.m. Vosoritide Safety and Effectiveness in Young Children With Achondroplasia Aged ≤3 Years and With up to 36 Months of Follow-Up from the Japanese Post-Marketing Safety Surveillance Study (111-604)Oral Presentation #FC3.4Tuesday, Sept. 8, 3:30 – 3:40 p.m. About Hypochondroplasia Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings. BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO. There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia. For more information about our clinical trials in hypochondroplasia, achondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com. About VOXZOGO In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures. VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). An sNDA with long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth such as body proportionality and arm span is under review with an FDA Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027. The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency. VOXZOGO U.S. Important Safety Information What is VOXZOGO used for? VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO? VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO? The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken? VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO? Tell your doctor about all of the patient's medical conditions including Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. Please see additional safety information in the full Prescribing Information and Patient Information. About BioMarin BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com. Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting, including the safety profile and potential benefits of VOXZOGO for children with hypochondroplasia and achondroplasia; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including the expectation that, if approved by the U.S. Food and Drug Administration (FDA), VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia with a potential 2027 launch; BioMarin's expectations regarding its supplemental New Drug Application (sNDA) for VOXZOGO for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; and BioMarin's estimate regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including hypochondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc. View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-positive-results-of-phase-3-voxzogo-vosoritide-study-in-hypochondroplasia-published-in-nejm-evidence-302873026.html

As the craze of earnings season draws to a close, here’s a look back at some of the most exciting (and some less so) results from Q2. Today, we are looking at therapeutics stocks, starting with BioMarin Pharmaceutical (NASDAQ:BMRN). Over the next few years, therapeutic companies, which develop a wide variety of treatments for diseases and disorders, face strong tailwinds from advancements in precision medicine (including the use of AI to improve hit rates) and growing demand for treatments targeting rare diseases. However, headwinds such as rising scrutiny over drug pricing, regulatory unknowns, and competition from larger, more resourced pharmaceutical companies could weigh on growth. The 11 therapeutics stocks we track reported a very strong Q2. As a group, revenues beat analysts’ consensus estimates by 8%. Luckily, therapeutics stocks have performed well with share prices up 22.5% on average since the latest earnings results. Pioneering treatments for conditions that often had no previous therapeutic options, BioMarin Pharmaceutical (NASDAQ:BMRN) develops and commercializes therapies that address the root causes of rare genetic disorders, particularly those affecting children. BioMarin Pharmaceutical reported revenues of $989.7 million, up 19.9% year on year. This print exceeded analysts’ expectations by 6.5%. Overall, it was a very strong quarter for the company with a beat of analysts’ full-year EPS guidance estimates. "This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin's operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin. Interestingly, the stock is up 13% since reporting and currently trades at $69.47. Is now the time to buy BioMarin Pharmaceutical? Access our full analysis of the earnings results here, it’s free. Founded in 1978 and pioneering treatments for some of medicine's most complex challenges, Biogen (NASDAQ:BIIB) develops and markets therapies for neurological conditions, including multiple sclerosis, Alzheimer's disease, spinal muscular atrophy, and rare diseases. Biogen reported revenues of $2.74 billion, up 3.4% year on year, outperforming analysts’ expectations by 12.1%. The business had an incredible quarter with a beat of analysts’ EPS estimates and a solid beat of analysts’ full-year EPS guidance estimates. The market seems happy with the results as the stock is up 6.9% since reporting. It currently trades at $219.81. Is now the time to buy Biogen? Access our full analysis of the earnings results here, it’s free. Founded in 1991 as one of the pioneers in translating genetic discoveries into clinical applications, Myriad Genetics (NASDAQ:MYGN) develops genetic tests that assess disease risk, guide treatment decisions, and provide insights across oncology, women's health, and mental health. Myriad Genetics reported revenues of $190.7 million, down 10.5% year on year, falling short of analysts’ expectations by 8.2%. It was a disappointing quarter as it posted full-year revenue guidance missing analysts’ expectations and a significant miss of analysts’ EPS estimates. Myriad Genetics delivered the weakest performance against analyst estimates and weakest full-year guidance update among its peers. As expected, the stock is down 41.8% since the results and currently trades at $3.13. Read our full analysis of Myriad Genetics’s results here. Known for transforming hours-long intravenous infusions into minutes-long subcutaneous injections, Halozyme Therapeutics (NASDAQ:HALO) develops and licenses its proprietary ENHANZE technology that enables subcutaneous delivery of injectable drugs that would otherwise require intravenous administration. Halozyme Therapeutics reported revenues of $481 million, up 47.7% year on year. This print topped analysts’ expectations by 19%. Overall, it was an incredible quarter as it also produced an impressive beat of analysts’ full-year EPS guidance estimates. Halozyme Therapeutics achieved the fastest revenue growth and highest full-year guidance raise in the group. The stock is up 25% since reporting and currently trades at $107.23. Read our full, actionable report on Halozyme Therapeutics here, it’s free. Founded in 1989 with a mission to create medicines that treat the underlying causes of disease rather than just symptoms, Vertex Pharmaceuticals (NASDAQ:VRTX) develops and markets transformative medicines for serious diseases, with a focus on cystic fibrosis, sickle cell disease, and pain management. Vertex Pharmaceuticals reported revenues of $3.33 billion, up 12.5% year on year. This number surpassed analysts’ expectations by 4.6%. It was a strong quarter as it also recorded full-year revenue guidance slightly topping analysts’ expectations. The stock is up 16.3% since reporting and currently trades at $547.25. Read our full, actionable report on Vertex Pharmaceuticals here, it’s free. Over the past year, investors have been forced to repeatedly answer the same question: what is the market’s biggest risk? The answer has changed several times, and each shift has reshaped market leadership. Late in 2025 and early 2026, artificial intelligence became the market’s primary uncertainty. Investors questioned whether AI would erode software pricing power and weaken competitive moats as AI made it easier to replicate once-differentiated products. By the spring, technology took a back seat to geopolitics. The U.S. conflict with Iran briefly became the market’s dominant narrative, raising concerns about oil prices, inflation, and global growth. But as energy markets remained orderly and fears of a prolonged supply disruption faded, investors quickly turned their focus back to fundamentals. Want to invest in winners with rock-solid fundamentals? Check out our Strong Momentum Stocks and add them to your watchlist. These companies are poised for growth regardless of the political or macroeconomic climate.

This article first appeared on GuruFocus. Total Revenue: Nearly $1 billion in Q2 2026, representing 20% year-over-year growth. Non-GAAP Operating Margin: 36.4% for the second quarter. Non-GAAP Diluted EPS: $1.20 for the second quarter. VOXZOGO Revenue: 14% year-over-year growth in Q2, driven by double-digit growth in both US and international markets. Metabolic Conditions Revenue: $695 million, up 25% year-over-year, including contributions from Galafold and PomBility/Opfolda. Galafold Revenue (Pro Forma): Approximately 10% year-over-year growth in Q2. PomBility and Opfolda Revenue (Pro Forma): Over 65% year-over-year growth in Q2. Non-GAAP R&D and SG&A Expenses: Increased year-over-year, reflecting operating expenses from the acquired Amicus business and continued investment in pipeline and commercial execution. Interest Expense: Increased year-over-year due to acquisition debt financing, estimated at approximately $200 million annualized, or about $50 million per quarter. Interest Income: Decreased as investments were liquidated to fund the acquisition. GAAP SG&A Charges: Included approximately $84 million in transaction and integration-related charges associated with the Amicus acquisition. Warning! GuruFocus has detected 3 Warning Signs with PRDO. Is BMRN fairly valued? Test your thesis with our free DCF calculator. Release Date: August 06, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. BioMarin Pharmaceutical Inc (NASDAQ:BMRN) delivered a standout second quarter with 20% year-over-year total revenue growth, reaching nearly $1 billion in revenue. The company successfully closed and integrated the Amicus acquisition, with Galafold and PomBility/Opfolda showing strong pro forma growth of approximately 10% and over 65% year-over-year, respectively. VOXZOGO demonstrated resilience despite a new US competitor, with 90% of US patients remaining on therapy and double-digit revenue growth, leading to increased full-year guidance and a path to become BioMarin's first $1 billion product. The company identified significant cost synergies of approximately $220 million annual run rate by 2028, expected to drive substantial EPS accretion and operating cash flow, with deleveraging accelerated by about one year. Pipeline momentum is strong, highlighted by the rapid SNDA submission for VOXZOGO in hypochondroplasia (79 days from database lock to filing) and the addition of BMN 820, a late-stage asset for FSGS with a favorable safety profile. The company raised full-year 2026 guidance for total revenues, VOXZOGO revenue, and non-GAAP diluted EPS, reflecting strong first-half performance and confidence in the second half. Galafold and PomBility/Opfolda have substantial peak revenue potential of $1.4 billion and $1.2 billion, respectively, supported by strategies to expand diagnosis, treatment, and global market penetration. Non-GAAP diluted EPS decreased year-over-year due to higher operating expenses from the Amicus acquisition, increased interest expense from acquisition debt financing, and lower interest income. The company faces competitive pressure in the US for VOXZOGO, with approximately 10% of US patients switching to the competitor product since its launch, and the full impact on future market share remains uncertain. The Amicus acquisition is expected to be modestly dilutive to non-GAAP EPS in 2026, with substantial accretion only beginning in 2027 and full synergies realized in 2028. Interest expense from acquisition debt financing is estimated at approximately $200 million annually, which will continue to weigh on non-GAAP EPS in the near term. The company faces ongoing legal and regulatory uncertainties, including the ITC case related to VOXZOGO patents, with a final decision expected in December 2026 and potential exclusion orders. Revenue in the metabolic conditions portfolio is subject to quarterly order timing fluctuations, which can create volatility in individual product revenues, as seen with Vimizim and Naglazyme. The company's growth plans for Galafold and PomBility/Opfolda rely on improving diagnosis rates and patient switching, which have historically been challenging in rare diseases like Fabry and Pompe, and may not materialize as quickly as projected. Q: Can you provide more detail on the mechanisms behind driving increased diagnosis and switching for Pombility/Opfolda and Galafold, and how do these efforts differ between the U.S. and ex-U.S.? Also, what is your appetite for future business development?A: Cristin Hubbard (Chief Commercial Officer) explained that the levers are similar across regions but executed at the country level. For Galafold, the focus is on expanding diagnosis, especially for late-onset and female Fabry patients, and closing the gap between diagnosis and treatment. For Pombility/Opfolda, the focus is on accelerating switches from current therapies by identifying clinically declining patients. Gregory Friberg (Chief R&D Officer) added that only about 40% of Fabry patients are diagnosed, and they are using electronic health records, family cascade testing, and variant reclassification to shorten diagnosis time. Alexander Hardy (CEO) stated that with the diversified portfolio, the focus is shifting to expanding the clinical-stage pipeline, and they expect to do deals over the next 12 to 18 months as they deleverage. Q: On VOXZOGO, can you give us a sense of the dynamic in terms of your competition for new patients, given the competitor's launch and the 90% retention rate you mentioned?A: Alexander Hardy (CEO) clarified that the competitor's enrollment numbers include naive patients, VOXZOGO discontinuations, and switches. According to BioMarin's data, approximately 10% of patients have switched, translating to less than 100 patient switches in about six months, which has a very small impact on revenue. He emphasized that new patient starts are dominated by the 0-2 age group, where BioMarin remains the only approved treatment. The strong growth, with a 20% increase in patients globally, led to raising VOXZOGO revenue guidance to over $1 billion for the year. Q: Regarding the Amicus integration, can you give some waypoints as we get through 2027 and tell us the specific steps you're taking to get to the $220 million synergy number and what's behind delivering the leverage target a year early?A: Brian Mueller (CFO) confirmed that the transaction is expected to be modestly dilutive in 2026 but close to breakeven in the first half. The company expects substantial accretion beginning in 2027, with 2028 being the first full year of realizing all synergy benefits. He guided that next year, they expect half to slightly more than half of the synergies to be realized. The integration plan is being executed end-to-end, and the decisions are already made, which is why they are confident in pulling forward the leverage target by approximately one year. Q: How do you think about the combination of weekly CNP analog plus growth hormone eventually slotting into the treatment algorithm, and is there anything stopping physicians from using VOXZOGO and/or BMN333 with growth hormone?A: Gregory Friberg (Chief R&D Officer) noted that the COACH study is small (about 21 patients) and should be interpreted cautiously. The effects of growth hormone adding on to CNP appear to be waning, and the key question is whether it will contribute to final adult height. There is a concern that growth hormone may close growth plates early, which cannot be assessed in an 18-month study. He stated that from a biologic standpoint, there is nothing unique about TransCon CNP when it comes to combining with growth hormone, and they are watching the data closely. Q: Do you expect the synergies associated with the Amicus deal to fall to the bottom line, or do you expect those to be reinvested in the business?A: Brian Mueller (CFO) confirmed that the synergies are expected to drop to the bottom line. However, there will be some reinvestment to accelerate the growth potential of Galafold and Pombility/Opfolda, which is a modest portion of the synergy amount. This reinvestment fits within the existing structure of the P&L and is part of normal metabolic conditions sales and marketing, so it is not considered an offset to the synergies themselves. Q: On the upcoming ITC case, we expect a decision by the end of August. Can you provide your most recent thoughts on that case and the possibility of a settlement? Also, why haven't late-onset and female Fabry patients been diagnosed so far despite past efforts?A: Alexander Hardy (CEO) outlined the ITC timeline: the administrative law judge will deliver an initial determination on August 21, with a final decision expected by December 21, followed by a presidential review period through February 21, 2027. He declined to speculate on outcomes or legal strategy. Gregory Friberg (Chief R&D Officer) explained that Fabry is an elusive disease with heterogeneous presentation across seven organ systems, leading to a 7-10 year diagnostic delay. He highlighted efforts using technology, electronic health records, and family cascade testing to shorten time to diagnosis, noting that finding patients early is key. Q: Of the 10% of patients in the US who switched from VOXZOGO, what trends or characteristics are you noticing in those patients versus those who stay on VOXZOGO? And what degree of switching is baked into guidance? Also, how should we think about the annual incidence of new starts in the under-2 age group?A: Cristin Hubbard (Chief Commercial Officer) stated that the primary reasons for switching are injection fatigue or wanting a weekly therapy. The 90% retention is driven by the surround-sound services, clinical coordinators, and the strong evidence base. She estimated about 150 births per year in the US for achondroplasia and emphasized targeting maternal-fetal medicine to treat as early as possible. Brian Mueller (CFO) noted that guidance included a switch assumption but declined to quantify it, citing the strong Q2 performance and confidence in the second half. Q: Regarding the 10% of patients who have switched, where do you expect this to settle in the US market? And how similar or different is the ex-US market in terms of structure for a competitor to take share?A: Cristin Hubbard (Chief Commercial Officer) said they are monitoring whether the switching is a bolus or steady state and declined to provide specific expectations. She noted that the US market is more segmented and geographically dispersed, with more specialties involved, which impacts competitive dynamics. Ex-US, there have been no competitor approvals yet, but she expects the dynamics to be similar, and they will remain vigilant. Q: Can you talk about what For the complete transcript of the earnings call, please refer to the full earnings call transcript.

BioMarin Pharmaceutical BMRN reported second-quarter 2026 adjusted earnings of $1.20 per share, which beat the Zacks Consensus Estimate of 96 cents. However, the bottom line declined 16.7% year over year from $1.44. Total revenues were $989.7 million, up 20% year over year, beating the Zacks Consensus Estimate of $922 million. Growth was driven by new Amicus-acquired products, Voxzogo patient additions and stronger Palynziq demand. Shares of BioMarin were up 4.4% in after-market trading on Thursday following the earnings announcement. The positive stock reaction came as both earnings and revenues topped expectations and management raised key elements of its 2026 financial guidance. Year to date, BMRN stock has gained 3.4% compared with the industry’s 3.6% growth. Image Source: Zacks Investment Research Net product revenues totaled nearly $984.4 million, up about 21% year over year, driven by higher revenues from the company’s Metabolic Conditions drugs, as well as Voxzogo. Voxzogo generated $253 million in sales, up 14% year over year from $221 million. Sales also beat the Zacks Consensus Estimate of $238.2 million. The number of children treated globally increased more than 20% year over year. BioMarin said about 90% of U.S. children treated with Voxzogo remained on therapy through the end of July after a competing product entered the market. More than half of new U.S. patient starts during the quarter were children under two years of age. Metabolic Conditions revenues totaled $695 million, up 25% year over year. The portfolio now comprises seven therapies, including Galafold and Pombiliti-Opfolda, which were added following the Amicus acquisition. Palynziq sales jumped 27% to $135 million, topping the Zacks Consensus Estimate of $112.7 million. Naglazyme revenues rose 5% to $135 million, which also came above the consensus estimate of $125.9 million. Brineura sales increased 4% to $51 million. Vimizim revenues fell 10% to $194 million, reflecting the timing of large government orders outside the United States. Sales missed the Zacks Consensus Estimate of $205.8 million. Aldurazyme sales declined 21% to $44 million due to timing of order fulfillment to Sanofi SNY. Galafold contributed $106 million following the Amicus acquisition. BioMarin said the therapy maintained broad-based patient growth, supported by increased diagnosis and patient identification. The Sanofi-related Aldurazyme order timing was a separate headwind within the portfolio. BioMarin signed a collaboration agreement with Sanofi’s subsidiary, Genzyme, for Aldurazyme. SNY, through Genzyme, is BMRN’s sole customer for Aldurazyme. The Sanofi subsidiary is responsible for marketing and selling Aldurazyme to third parties. Kuvan generated $24 million in second-quarter sales, down 11% from $27 million a year earlier. Roctavian revenues were $12 million, up 33% year over year from $9 million. Royalty and other revenues were $5.3 million compared with $12.4 million in the year-ago quarter. BioMarin raised its 2026 total revenue guidance to $3.875-$3.925 billion from $3.825-$3.925 billion. Metabolic Conditions revenue guidance was maintained at $2.725-$2.775 billion. Voxzogo revenue guidance was increased to $1-$1.05 billion from $975-$1.025 billion. Other revenues are expected to be in the range of $100-$125 million in 2026, unchanged from the previous guidance. Adjusted earnings guidance was raised to $4.90-$5.10 per share from $4.85-$5.05. BioMarin submitted a supplemental new drug application (NDA) to the FDA seeking approval of Voxzogo for hypochondroplasia after the phase III CANOPY-HCH-3 study met its primary endpoint. The company expects to provide an update on the application status with its third-quarter earnings update. The FDA also accepted BioMarin's supplemental NDA for full approval of Voxzogo in children with achondroplasia, with a Feb. 28, 2027, target action date. Meanwhile, the company discontinued BMN 401 after the ENERGY 3 study failed to meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency. A data update from the phase II/III study of BMN 333 for achondroplasia is expected in 2027. In the second quarter, the European Commission approved Palynziq for adolescents aged 12 years and older with phenylketonuria (PKU). The label expansion broadens access to Palynziq, which enables patients with PKU to achieve physiologic phenylalanine levels while reducing dietary restrictions, regardless of disease severity. BioMarin Pharmaceutical Inc. price-consensus-eps-surprise-chart | BioMarin Pharmaceutical Inc. Quote BioMarin's quarter showed broad commercial momentum, with Voxzogo growth and the newly acquired Galafold contribution supporting a strong revenue beat. Palynziq and Naglazyme also exceeded expectations, while Vimizim and Aldurazyme faced order-timing pressure. The raised outlook adds to the positive read-through from the quarter, though product-level volatility remains evident. In particular, Aldurazyme's decline reflected order fulfillment timing to Sanofi, making SNY-related ordering an important factor to watch alongside competitive dynamics in Voxzogo. BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Harmony Biosciences HRMY and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here. Over the past 60 days, estimates for Harmony Biosciences’ 2026 earnings per share have risen from $3.20 to $3.33, while estimates for 2027 have increased from $3.64 to $3.92 during the same time. HRMY shares have gained 3.5% year to date. Harmony Biosciences’ earnings missed estimates in three of the trailing four quarters and beat on the remaining occasion, delivering an average negative surprise of 13.97%. Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $2.97 to $3.02, while estimates for 2027 have increased from $4.81 to $5.31 during the same time. LQDA shares have surged 159.3% year to date. Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report Sanofi (SNY) : Free Stock Analysis Report Liquidia Corporation (LQDA) : Free Stock Analysis Report Harmony Biosciences Holdings, Inc. (HRMY) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

BioMarin Pharmaceutical (BMRN) came out with quarterly earnings of $1.2 per share, beating the Zacks Consensus Estimate of $0.96 per share. This compares to earnings of $1.44 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of +25.00%. A quarter ago, it was expected that this rare disease biopharmaceutical would post earnings of $0.94 per share when it actually produced earnings of $0.76, delivering a surprise of -19.15%. Over the last four quarters, the company has surpassed consensus EPS estimates three times. BioMarin, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $989.71 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 7.31%. This compares to year-ago revenues of $825.41 million. The company has topped consensus revenue estimates three times over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. BioMarin shares have added about 0.8% since the beginning of the year versus the S&P 500's gain of 12.8%. While BioMarin has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for BioMarin was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $1.49 on $1.04 billion in revenues for the coming quarter and $4.95 on $3.86 billion in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 44% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. BioXcel Therapeutics, Inc. (BTAI), another stock in the same industry, has yet to report results for the quarter ended June 2026. This company is expected to post quarterly loss of $0.51 per share in its upcoming report, which represents a year-over-year change of +79.2%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. BioXcel Therapeutics, Inc.'s revenues are expected to be $0.34 million, up 183.3% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report BioXcel Therapeutics, Inc. (BTAI) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

BioMarin Pharmaceutical (BMRN) reported $989.71 million in revenue for the quarter ended June 2026, representing a year-over-year increase of 19.9%. EPS of $1.20 for the same period compares to $1.44 a year ago. The reported revenue represents a surprise of +7.31% over the Zacks Consensus Estimate of $922.33 million. With the consensus EPS estimate being $0.96, the EPS surprise was +25%. While investors closely watch year-over-year changes in headline numbers -- revenue and earnings -- and how they compare to Wall Street expectations to determine their next course of action, some key metrics always provide a better insight into a company's underlying performance. As these metrics influence top- and bottom-line performance, comparing them to the year-ago numbers and what analysts estimated helps investors project a stock's price performance more accurately. Here is how BioMarin performed in the just reported quarter in terms of the metrics most widely monitored and projected by Wall Street analysts: Revenues- Net product revenues: $984.39 million versus the eight-analyst average estimate of $914.12 million. The reported number represents a year-over-year change of +21.1%. Revenues- Royalty and other revenues: $5.32 million compared to the $8.21 million average estimate based on eight analysts. The reported number represents a change of -57.2% year over year. Revenues- Net Product Revenues- NAGLAZYME: $135 million compared to the $125.91 million average estimate based on seven analysts. The reported number represents a change of +4.7% year over year. Revenues- Net Product Revenues- VIMIZIM: $194 million compared to the $205.82 million average estimate based on seven analysts. The reported number represents a change of -9.8% year over year. Revenues- Net Product Revenues- PALYNZIQ: $135 million versus $112.75 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +27.4% change. Revenues- Net Product Revenues- VOXZOGO: $253 million versus the seven-analyst average estimate of $238.2 million. The reported number represents a year-over-year change of +14.5%. Revenues- Net Product Revenues- KUVAN: $24 million versus the seven-analyst average estimate of $17.25 million. The reported number represents a year-over-year change of -11.1%. Revenues- Net Product Revenues- ALDURAZYME: $44 million versus the seven-analyst average estimate of $52.41 million. The reported number represents a year-over-year change of -21.4%. Revenues- Net Product Revenues- BRINEURA: $51 million versus $50.06 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +4.1% change. View all Key Company Metrics for BioMarin here>>> Shares of BioMarin have returned +0.3% over the past month versus the Zacks S&P 500 composite's +3.3% change. The stock currently has a Zacks Rank #3 (Hold), indicating that it could perform in line with the broader market in the near term. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

Interested in BioMarin Pharmaceutical Inc.? Here are five stocks we like better. BioMarin’s second-quarter revenue nearly reached $1 billion, rising 20% year over year. The company raised its full-year revenue, VOXZOGO revenue and non-GAAP EPS outlook following strong rare-disease portfolio growth and the Amicus acquisition. Amicus therapies are expected to significantly expand BioMarin’s long-term growth. The company projects peak revenue of $1.4 billion for GALAFOLD and $1.2 billion for POMBILITI/OPFOLDA, with $220 million in annual cost synergies expected by 2028; the deal should become substantially earnings-accretive in 2027. VOXZOGO continued to grow despite new U.S. competition. Revenue increased 14%, global treated children rose more than 20%, and BioMarin said about 90% of U.S. patients remained on therapy while the drug stayed on track to become its first $1 billion product. Can BioMarin Stock Live Up to Wall Street’s High Expectations? BioMarin Pharmaceutical (NASDAQ:BMRN) reported second-quarter 2026 revenue of nearly $1 billion, up 20% from a year earlier, as growth across its rare-disease portfolio and the recently completed Amicus acquisition supported results. The company raised its full-year revenue, VOXZOGO revenue and non-GAAP diluted earnings-per-share guidance, though it did not provide updated ranges during the call. Chief Executive Officer Alexander Hardy said the quarter combined strong commercial execution with the close and integration of Amicus. BioMarin expects the acquired GALAFOLD and POMBILITI and OPFOLDA therapies to expand its growth profile through the mid-2030s, supported by international expansion, patient identification efforts and planned cost savings. → 3 Drone Stocks That Should Soar After the Summer Slump 3 Oversold Stocks with Big RSI Rebound Potential BioMarin projected peak revenue of $1.4 billion for GALAFOLD, a treatment for Fabry disease, and $1.2 billion for POMBILITI and OPFOLDA, a combination therapy for Pompe disease. The company expects GALAFOLD revenue to grow at an approximately 10% compound annual rate from 2027 through 2032, while it expects POMBILITI and OPFOLDA to grow at a rate of at least 20% over the same period. On a pro forma basis, GALAFOLD revenue increased about 10% year over year in the second quarter, while POMBILITI and OPFOLDA revenue rose more than 65%, according to Chief Commercial Officer Cristin Hubbard. GALAFOLD’s growth was driven by patient additions in established and newer markets, while POMBILITI and OPFOLDA added patients in the U.S. and recently launched geographies. → Meta’s Earnings Drop Shows Wall Street Wants More Than Ad Growth Are Gene Therapy Stocks The Market's Next Big Winners? The company expects approximately $220 million in annual run-rate cost synergies to be fully realized in 2028, representing about a 50% reduction from Amicus’ 2025 non-GAAP operating expenses of $432 million. More than 70% of the expected savings are expected to come from general and administrative expenses, with the remaining savings primarily from research and development. Chief Financial Officer Brian Mueller said the acquisition is still expected to be modestly dilutive in calendar 2026, though it is “close to breakeven.” BioMarin expects substantial earnings accretion to begin in 2027, with roughly half to slightly more than half of the planned synergies expected to be realized next year. The company also expects to reduce leverage about one year earlier than previously communicated. → Jersey Mike's Serves Fresh Gains After IPO Stumble Mueller said acquisition-related debt is expected to generate annualized interest expense of roughly $200 million, or about $50 million per quarter, based on current rates. That expense is included in non-GAAP results. Interest income is also expected to decline in the near term following the use of cash and investments to fund the acquisition. VOXZOGO revenue grew 14% year over year in the second quarter, with double-digit growth in both the U.S. and international markets. The number of children receiving the treatment increased more than 20% globally from a year earlier, and approximately three-quarters of VOXZOGO revenue came from outside the U.S. BioMarin said it raised its full-year VOXZOGO outlook following first-half performance and expectations for the remainder of 2026. Mueller said one international pricing negotiation closed with a favorable outcome, while another remained in process after initial setbacks. Growth in both the U.S. and global markets also contributed to the improved outlook. The company is managing competition in the U.S. achondroplasia market following a competitor’s February launch. Hubbard said that approximately 90% of U.S. children using VOXZOGO had remained on treatment as of the end of July, based on the company’s available information. Hardy said BioMarin had observed approximately 10% of U.S. VOXZOGO patients switching, or fewer than 100 patients over roughly six months. Hubbard said patients who switched were primarily seeking less frequent dosing or responding to injection fatigue. BioMarin said more than half of its new U.S. patient starts during the quarter were in children ages two and younger, an age group for which it said VOXZOGO remains the only approved treatment. Chief Research and Development Officer Greg Friberg estimated there are about 150 U.S. births annually involving infants with achondroplasia. BioMarin expects both patient additions and ordering patterns to lift VOXZOGO revenue in the second half compared with the first half. The company said VOXZOGO is on track to become its first $1 billion product. BioMarin submitted a supplemental new drug application for VOXZOGO in hypochondroplasia, following pivotal data reported during the quarter. Friberg said the company reduced the time from database lock to filing to 79 days through parallel work processes and technology-enabled efforts. Full Phase III results are scheduled for presentation at the ESPE meeting in September, and BioMarin plans to provide an update on the filing with third-quarter results. The company estimates a global addressable hypochondroplasia population of approximately 14,000 patients. BioMarin said it is pursuing physician education, digital campaigns, genetic testing and other patient-identification initiatives ahead of a potential launch. The Amicus transaction also added BMN 820, formerly DMX-200, to BioMarin’s pipeline. The oral CCR2 inhibitor is in Phase III development for focal segmental glomerulosclerosis, or FSGS. BioMarin holds exclusive U.S. commercialization rights, while partner Dimerix is responsible for operating the Phase III ACTION 3 study. The company expects Phase III data in 2028. BioMarin’s Metabolic Conditions business, formerly called Enzyme Therapies, generated $695 million in second-quarter revenue, up 25% year over year including the acquired Amicus products. PALYNZIQ revenue increased 27%, aided by patient demand and U.S. ordering timing. The company also recently received European approval to expand PALYNZIQ’s label to adolescents ages 12 and older with PKU. For the second half, Mueller said third-quarter revenue should be slightly above the second quarter, reflecting a full quarter of Amicus contributions. He expects the fourth quarter to be the company’s strongest of 2026, with ordering dynamics in select markets accounting for well over half of projected second-half revenue. BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe. The company's commercial portfolio includes several approved therapies targeting inherited disorders. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "BioMarin Pharmaceutical Q2 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for August 2026.

Second Quarter 2026 Total Revenues Increased 20% Year-over-year to $990 million Stronger Growth Expectations Drive Increased Guidance for Full-year 2026 Total Revenues, VOXZOGO®, and Non-GAAP Diluted Earnings Per Share (EPS) Addition of GALAFOLD® and POMBILITI® + OPFOLDA®, with Cost Synergies, Expected to Accelerate Revenue Growth, Non-GAAP Diluted EPS Accretion, Non-GAAP Operating Margin Expansion, and Operating Cash Flow through the Mid-2030s Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., Aug. 6, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the second quarter ended June 30, 2026. "This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin's operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026. Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations." Mr. Hardy added, "With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability. We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world." 2026 Business and Pipeline Highlights Innovation BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch. The company plans to provide an update on the application status as part of its third quarter earnings update. In May, the company announced that the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity (AGV) at week 52 versus placebo (LS mean difference +2.33 cm/yr, p In June, at the Endocrine Society Annual Meeting (ENDO 2026), a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p Also at ENDO 2026, the company presented Phase 1 data for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a single-ascending-dose study in healthy adults, BMN 333 demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent, reflecting its potential to become a new standard of care in achondroplasia. The Phase 2/3 study is enrolling, with a data update expected in 2027. In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027. In the second quarter, the European Commission approved PALYNZIQ® for adolescents 12 years and older with phenylketonuria (PKU). PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. During the quarter, BioMarin added BMN 820 (formerly DMX-200) to its portfolio, a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS) for which BioMarin holds exclusive U.S. commercialization rights. BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000. The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028. BMN 351, BioMarin's Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development. The company expects to provide a program update by year-end. Following the pivotal ENERGY 3 trial results, previously announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications. In July, BioMarin and the n-Lorem Foundation entered a collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. ReNU syndrome has an expected global population of approximately 100,000. Growth BioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s and for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s. BioMarin expects these high growth therapies to benefit from its global scale and proven commercial capabilities. Metabolic Conditions (formerly Enzyme Therapies) revenue grew 25% Y/Y in the second quarter of 2026, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ. The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially. Strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion. The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter. In the U.S., the majority of new patient starts were under two years of age, and the region drove approximately 25% of total VOXZOGO revenue during the quarter. Value Commitment As part of the acquisition of Amicus, which closed on April 27, 2026, the company identified approximately $280 million of cost reductions on a GAAP basis, and approximately $220 million of cost reductions on a Non-GAAP basis, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. Synergies reflect a reduction of Amicus' legacy labor costs and external spend and are expected to be largely driven by general and administrative functions, with the large majority of sales and marketing capabilities retained to support continued commercial growth. GALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030. The company is targeting gross leverage below 2.5 times by mid-year 2027, an acceleration by approximately one year of prior timeline guidance provided at deal announcement, supported by profitability growth of the combined company. Second Quarter 2026 Financial Highlights Total Revenues for the second quarter of 2026 were $990 million, an increase of $165 million compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired from Amicus on April 27, 2026, as well as new patients initiating VOXZOGO therapy across all regions and growth in U.S. patients treated with PALYNZIQ. These increases were partially offset by lower VIMIZIM® revenue due to the timing of large government orders outside the U.S. and lower ALDURAZYME® sales volume due to the timing of order fulfillment to Sanofi. GAAP Net Income for the second quarter of 2026 decreased to $45 million compared to $241 million for the same period in 2025. The decrease was primarily driven by the acquisition of Amicus, including integration and restructuring costs, intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, and amortization of inventory fair value step-up. Other drivers included higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO and higher Research and Development (R&D) spend related to BMN 401, which was acquired in the third quarter of 2025, partially offset by higher gross profit driven by revenue growth as described above. Non-GAAP Income for the second quarter of 2026 decreased to $236 million compared to $282 million for the same period in 2025. The decrease was primarily driven by higher interest expense, higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO, and higher R&D spend related to BMN 401, partially offset by higher gross profit driven by revenue growth as described above. Forward-Looking Non-GAAP Financial Information BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors. Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts) Total Revenues, VOXZOGO, and Non-GAAP Diluted EPS guidance raised, reflecting strong first-half 2026 performance and second-half 2026 revenue expectations for both Metabolic Conditions and VOXZOGO. Guidance reflects post-close contributions from Amicus beginning April 27, 2026. BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non‑GAAP financial results. Based on current rates, interest expense associated with the financing is estimated at approximately $200 million on an annualized basis, with Term Loans and Senior Notes scheduled to mature after 2030. BioMarin will host a conference call and webcast to discuss second quarter 2026 financial results today, Thursday, August 6, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com. About BioMarin BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com. Forward-Looking Statements This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Diluted EPS, Non-GAAP Operating Margin, gross leverage, operating cash flow and revenue compound annual growth rate (CAGR) for, in certain instances, the full-year 2026, fourth quarter and second half of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin's Metabolic Conditions portfolio, including PALYNZIQ, and VOXZOGO, and the expected impact of the acquisition of Amicus Therapeutics, Inc. (Amicus); the anticipated benefits of the acquisition of Amicus, including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA, including BioMarin's plans and expectations to accelerate growth through mid-2030s; BioMarin's plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin's product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin's commercial products and product candidates, including with respect to the potential new indication for VOXZOGO in hypochondroplasia; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; BioMarin's ability to realize the anticipated benefits of any acquisitions; BioMarin's ability to accurately estimate future financial performance; impacts of macroeconomic and other external factors on BioMarin's operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; geopolitical instability, wars and military conflicts; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin's ability to meet product demand; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN®, ROCTAVIAN®, GALAFOLD®, and POMBILITI® + OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners. Non-GAAP Information The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Cost of Sales (COS), Non-GAAP R&D expenses and Non-GAAP Selling, General and Administrative (SG&A) expenses are defined by the company as GAAP COS, GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans or convertible debt in periods when they are dilutive under Non-GAAP. Projected Gross Leverage is defined by the company as undiscounted debt (total debt excluding unamortized discount and deferred offering costs) as of the balance sheet date divided by 4 quarter projected Non-GAAP Adjusted EBITDA. Non-GAAP Adjusted EBITDA is defined by the company as GAAP Income (or Loss) from Operations excluding the impact of depreciation, amortization and stock-based compensation expense. Non-GAAP synergies is defined by the company as Amicus' legacy labor and external spend cost reductions, excluding the impact of stock-based compensation. BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Because these Non-GAAP metrics are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business. Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information: View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-reports-second-quarter-2026-financial-and-operating-results-302845167.html