BMRN

Data from an investigator-led study showed treatment with VOXZOGO resulted in sustained improvements in growth over three years in children with hypochondroplasia New Phase 1 results also presented for investigational BMN 333 support potential weekly dosing in children with achondroplasia SAN RAFAEL, Calif., June 16, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced new data from studies of VOXZOGO® (vosoritide) in children with hypochondroplasia and the investigational medicine BMN 333 in achondroplasia were presented at ENDO 2026, the Endocrine Society Annual Meeting, in Chicago. New data from a Phase 2, investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. and investigators at Children's National Hospital, demonstrated sustained improvements in annualized growth velocity (AGV) and height standard deviation score (SDS), with a favorable safety profile. Mean height SDS improved by 0.72 SD over three years of treatment, while mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through years two and three. BioMarin recently announced positive topline results from CANOPY-HCH-3, its registration-enabling Phase 3 pivotal study evaluating VOXZOGO in children with hypochondroplasia. These results will be included in the supplemental New Drug Application submission to the U.S. Food and Drug Administration planned for the third quarter of 2026. "Building on the excellent Phase 3 results we recently announced, these new longer-term data further reinforce the potential of VOXZOGO to meaningfully improve growth outcomes for children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Importantly, we continue to observe sustained growth improvements over time and a favorable safety profile, adding to the growing body of evidence supporting VOXZOGO for this potential new indication." New BMN 333 Phase 1 Data Support Advancement Into Late-Stage Development Additional data being presented at ENDO 2026 highlight the potential of investigational BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a Phase 1 single-ascending dose study in healthy adults, BMN 333 demonstrated sustained systemic exposure asso...

BioMarin Pharmaceutical (BMRN) said Tuesday it presented new clinical data for its Voxzogo and BMN 3

A month has gone by since the last earnings report for BioMarin Pharmaceutical (BMRN). Shares have added about 0.2% in that time frame, underperforming the S&P 500. Will the recent positive trend continue leading up to its next earnings release, or is BioMarin due for a pullback? Well, first let's take a quick look at the latest earnings report in order to get a better handle on the recent drivers for BioMarin Pharmaceutical Inc. before we dive into how investors and analysts have reacted as of late. BioMarin reported first-quarter 2026 adjusted earnings per share of 76 cents, missing the Zacks Consensus Estimate of 94 cents. However, earnings declined 33% year over year. This was largely due to a $31 million charge tied to the company’s unsuccessful campaign to extend Naglazyme manufacturing capabilities, as well as higher operating expenses associated with the acquisition of Amicus Therapeutics. Total revenues in the first quarter were $766.2 million, up 3% year over year. The figure beat the Zacks Consensus Estimate of $762.4 million. Net product revenues totaled nearly $760.1 million, up 3.5% year over year on higher revenues from the company’s Enzyme Therapies, as well as Voxzogo. Royalty and other revenues totaled $6.1 million, down about 42% year over year. Voxzogo generated sales of $220 million, up 3% year over year. Per the company, this modest upside was expected, as it had previously experienced large orders for the drug in the fourth quarter of 2025. Despite this, Voxzogo sales beat the Zacks Consensus Estimate of $216 million. BioMarin reports consolidated revenues from five products — Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim — under a single segment, “Enzyme Therapies.” Sales from this franchise increased 6% year over year to $514 million in the reported quarter, driven by higher product sales of Vimizim, Naglazyme and Brineura. Palynziq injection sales totaled $90 million in the quarter, down 3% year over year, impacted by order timing in the United States. The drug’s sales missed the Zacks Consensus Estimate of $112 million. Vimizim sales rose 12% year over year to $210 million, which beat the Zacks Consensus Estimate of $194 million. Naglazyme sales increased 14% year over year to $130 million. Brineura generated sales of $47 million, up 18%. Product revenues from Aldurazyme totaled $37 million, down 24% year over year. Roctavi...

Earlier this week, BioMarin Pharmaceutical reported mixed pivotal Phase 3 ENERGY 3 results for BMN 401 in children with ENPP1 deficiency, showing a statistically significant rise in plasma PPi but no improvement in key skeletal clinical endpoints or radiographic measures of rickets. The outcome highlights the challenge of translating promising biomarker changes into measurable clinical benefit, a core consideration for rare disease drug development and regulatory assessment. Next, we’ll explore how BMN 401’s biomarker success but lack of skeletal improvement may influence BioMarin’s broader investment narrative. Capitalize on the AI infrastructure supercycle with our selection of the 44 best 'picks and shovels' of the AI gold rush converting record-breaking demand into massive cash flow. To own BioMarin, you need to be comfortable with a focused rare-disease model where a handful of therapies and late-stage assets drive most of the value. The mixed BMN 401 Phase 3 readout increases attention on regulatory risk and the company’s ability to convert biomarker wins into clear clinical outcomes, but the most immediate catalyst and risk profile still center on VOXZOGO and other commercial products rather than BMN 401 alone. Against that backdrop, BioMarin’s decision on 4 May 2026 to raise full-year revenue guidance to US$3,825 million to US$3,925 million stands out. It shows that, prior to the BMN 401 update, management was confident enough in VOXZOGO, enzyme therapies, and recent approvals such as PALYNZIQ in adolescents to lift expectations, which now sit in sharper contrast to the heightened trial and regulatory risk investors are weighing after ENERGY 3. Yet, even if near term guidance holds, investors should be aware that... Read the full narrative on BioMarin Pharmaceutical (it's free!) BioMarin Pharmaceutical's narrative projects $4.9 billion revenue and $1.3 billion earnings by 2029. Uncover how BioMarin Pharmaceutical's forecasts yield a $89.96 fair value, a 79% upside to its current price. Some of the lowest ranked analysts were already cautious, assuming revenue of about US$4.8 billion and earnings of US$1.1 billion by 2029, and the BMN 401 setback could further reinforce their concern about heavy reliance on a few high priced rare disease drugs and the risk that clinical or regulatory disappointments derail the growth story. Explore 4 other fair value...

Study met primary endpoint, exceeding expectations with a highly statistically significant improvement in annualized growth velocity (AGV, change from baseline) of 2.33 cm/yr compared to placebo at week 52, as well as statistically significant increases in standing height and height Z-score Study demonstrated statistically significant improvement in arm span at week 52 compared to placebo, a key measure linked to functional independence Safety findings consistent with the established profile in achondroplasia with no new safety signals identified Supplemental New Drug Application (sNDA) submission to U.S. Food and Drug Administration (FDA) planned for third quarter, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities Full data to be presented at an upcoming medical meeting SAN RAFAEL, Calif., May 20, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO® (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualized growth velocity (AGV) compared to placebo (LS mean difference +2.33 cm/yr, p<0.0001). Children who received VOXZOGO also showed a statistically significant increase in standing height (p<0.0001) and height Z-score (p<0.0001) versus placebo after one year of treatment. Additionally, significant improvements in arm span were demonstrated (p=0.004), a key prespecified secondary endpoint in the study. These positive findings could have important implications for children with hypochondroplasia, offering improvements in reach, daily activities and independence, which have been highlighted as meaningful outcomes by the community. "As someone who treats children with hypochondroplasia, I find these positive results tremendously encouraging. Seeing these improvements in growth is a milestone we have hoped for after so many years without treatment options," said Dr. Andrew Dauber, who is the lead study investigator and Chief of Endocrinology at Children's National Hospital in Washington, D.C. "These data suggest we may be approaching a new era in how we care for children with hypochondroplasia." "For the first time, a pivotal Phase 3 study in hypochondroplasia has demonstrated impressive gains in gr...

Shares of BioMarin Pharmaceutical BMRN were down 4% on Monday after it reported results from the phase III ENERGY 3 study, which evaluated BMN 401, an investigational enzyme replacement therapy (ERT), for a rare genetic disorder called ENPP1 deficiency in children aged 1 to 12. The study did not meet one of its two main goals. The mixed results were a setback for BioMarin, which added BMN 401 through its $270 million acquisition of Inozyme Pharma last year. This ERT was the lead asset in Inozyme’s pipeline, and the ENERGY 3 study was already underway at the time of the acquisition. Year to date, the stock has lost 16% compared with the industry’s 3% fall. Image Source: Zacks Investment Research The study assessed two primary endpoints — changes in plasma inorganic pyrophosphate (PPi) and Radiographic Global Impression of Change (RGI-C) scores after 52 weeks of treatment. While treatment with BMN 401 after 52 weeks resulted in statistically significant increases in plasma PPi, there was no corresponding improvement in RGI-C scores. An increase in plasma PPi is considered an important biomarker response because ENPP1 deficiency leads to low PPi levels, which can result in progressive damage to blood vessels, soft tissues and bones. However, BMN 401 failed to demonstrate improvement in RGI-C scores, a key measure used to assess treatment impact in children with rickets. The lack of radiographic improvement suggests that the biomarker gains did not translate into measurable skeletal benefits during the study period. The therapy also did not show positive trends across secondary endpoints, including Rickets Severity Score (RSS), a measure of rickets severity, and growth Z-scores evaluating height/body length and weight. BioMarin stated that it will continue analyzing the complete ENERGY 3 dataset and engage with global regulatory authorities to determine the next steps for BMN 401’s development program. The company also plans to present detailed findings from the study at a future medical meeting. The stock currently carries a Zacks Rank #3 (Hold). BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote Some better-ranked stocks from the sector are Immunocore IMCR and Indivior Pharmaceuticals INDV, 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 30 days, estima...

BioMarin’s first quarter saw revenue growth driven by continued demand for its enzyme therapies and Voxzogo, but the company missed Wall Street’s revenue and non-GAAP profit expectations. Management cited strong patient demand, especially for newly approved indications like adolescent Palynziq, but also acknowledged that order timing, elevated costs from manufacturing issues, and integration costs from the Amicus acquisition weighed on margins. CFO Brian Mueller described the quarter’s profitability as “significantly impacted” by these factors, noting that “the cost of sales charge and pre-close costs associated with the Amicus acquisition resulted in a $0.20 earnings per share impact.” Is now the time to buy BMRN? Find out in our full research report (it’s free). Revenue: $766.2 million vs analyst estimates of $776 million (2.8% year-on-year growth, 1.3% miss) Adjusted EPS: $0.76 vs analyst expectations of $0.91 (16.9% miss) Adjusted EBITDA: $189.5 million vs analyst estimates of $150.8 million (24.7% margin, 25.6% beat) The company lifted its revenue guidance for the full year to $3.88 billion at the midpoint from $3.38 billion, a 14.8% increase Management lowered its full-year Adjusted EPS guidance to $4.95 at the midpoint, a 2% decrease Operating Margin: 16.9%, down from 30% in the same quarter last year Market Capitalization: $10.45 billion While we enjoy listening to the management's commentary, our favorite part of earnings calls are the analyst questions. Those are unscripted and can often highlight topics that management teams would rather avoid or topics where the answer is complicated. Here is what has caught our attention. Mike (Morgan Stanley) asked about bone health benefits in hypochondroplasia and expectations for upcoming data; Chief R&D Officer Gregory Friberg said the biology supports strong outcomes and expects to share results in Q2. Tommie Reerink (Goldman Sachs) inquired about competitive impacts on Voxzogo; Chief Commercial Officer Cristin Hubbard reported strong Q1 demand and reduced time to treatment, especially in children under two. Paul Andrew Matteis (Stifel) questioned the timing and scope of revenue and synergy updates post-Amicus; CFO Brian Mueller confirmed more details—including peak revenue and accretion—will be provided next quarter. Jasmine Fels (J.P. Morgan) asked about adoption rates for Voxzogo in hypochondroplasia an...

BioMarin Pharmaceutical BMRN reported first-quarter 2026 adjusted earnings per share of 76 cents, missing the Zacks Consensus Estimate of 94 cents. However, earnings declined 33% year over year. This was largely due to a $31 million charge tied to the company’s unsuccessful campaign to extend Naglazyme manufacturing capabilities, as well as higher operating expenses associated with the acquisition of Amicus Therapeutics. Total revenues in the first quarter were $766.2 million, up 3% year over year. The figure beat the Zacks Consensus Estimate of $762.4 million. Shares of BioMarin were down in after-hours trading on Monday, likely due to the mixed earnings results. Year to date, the stock has lost about 7% compared with the industry’s 2% decline. Image Source: Zacks Investment Research Net product revenues totaled nearly $760.1 million, up 3.5% year over year on higher revenues from the company’s Enzyme Therapies, as well as Voxzogo. Royalty and other revenues totaled $6.1 million, down about 42% year over year. Voxzogo, approved for achondroplasia, generated sales of $220 million, up 3% year over year. Per the company, this modest upside was expected, as it had previously experienced large orders for the drug in the fourth quarter of 2025. Despite this, Voxzogo sales beat the Zacks Consensus Estimate of $216 million. BioMarin reports consolidated revenues from five products — Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim — under a single segment, “Enzyme Therapies.” Sales from this franchise increased 6% year over year to $514 million in the reported quarter, driven by higher product sales of Vimizim, Naglazyme and Brineura. Palynziq injection sales totaled $90 million in the quarter, down 3% year over year, impacted by order timing in the United States. The drug’s sales missed the Zacks Consensus Estimate of $112 million. Vimizim sales rose 12% year over year to $210 million, which beat the Zacks Consensus Estimate of $194 million. Naglazyme sales increased 14% year over year to $130 million. Brineura generated sales of $47 million, up 18%. Product revenues from Aldurazyme totaled $37 million, down 24% year over year. BioMarin signed a collaboration agreement with Sanofi’s SNY 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...

BioMarin Pharmaceutical (BMRN) came out with quarterly earnings of $0.76 per share, missing the Zacks Consensus Estimate of $0.94 per share. This compares to earnings of $1.13 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -18.96%. A quarter ago, it was expected that this rare disease biopharmaceutical would post earnings of $0.25 per share when it actually produced earnings of $0.46, delivering a surprise of +84%. 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 $766.21 million for the quarter ended March 2026, surpassing the Zacks Consensus Estimate by 0.50%. This compares to year-ago revenues of $745.15 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 lost about 9% since the beginning of the year versus the S&P 500's gain of 5.6%. 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 t...

For the quarter ended March 2026, BioMarin Pharmaceutical (BMRN) reported revenue of $766.21 million, up 2.8% over the same period last year. EPS came in at $0.76, compared to $1.13 in the year-ago quarter. The reported revenue represents a surprise of +0.5% over the Zacks Consensus Estimate of $762.4 million. With the consensus EPS estimate being $0.94, the EPS surprise was -18.96%. 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- ALDURAZYME: $37 million versus $46.11 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a -24.5% change. Revenues- Net Product Revenues- KUVAN: $24 million versus $15.5 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a -4% change. Revenues- Net Product Revenues- NAGLAZYME: $130 million versus $117.67 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +14% change. Revenues- Net Product Revenues- VIMIZIM: $210 million compared to the $193.58 million average estimate based on seven analysts. The reported number represents a change of +11.7% year over year. Revenues- Royalty and other revenues: $6.13 million compared to the $15.58 million average estimate based on seven analysts. The reported number represents a change of -41.6% year over year. Revenues- Net Product Revenues- PALYNZIQ: $90 million compared to the $111.91 million average estimate based on seven analysts. The reported number represents a change of -3.2% year over year. Revenues- Net Product Revenues- VOXZOGO: $220 million versus the seven-analyst average estimate of $216.32 million. The reported number represents a year-over-year change of +2.8%. Revenues- Net product revenues: $514 million compared to the $746...

Image source: The Motley Fool. May 4, 2026 at 4:30 p.m. ET Chief Executive Officer — Alexander Hardy Chief Financial Officer — Brian Mueller Chief Commercial Officer — Cristin Hubbard Chief Research & Development Officer — Gregory Friberg joining today's call are Alexander Hardy, chief executive officer; Brian Mueller, chief financial officer; Cristin Hubbard, chief commercial officer; and Gregory Friberg, chief R&D officer. I will now turn the call over to BioMarin Pharmaceutical Inc.'s President and CEO, Alexander Hardy. Alexander Hardy: Thank you, Traci, and thank you all for joining us today. I am so pleased that we completed the Amicus acquisition last week, starting a new and exciting chapter for BioMarin Pharmaceutical Inc. with the addition of two innovative therapies, Galafold for Fabry disease and Pommability and Unfolda for Pompe disease. The acquisition accelerates our anticipated year-over-year 2026 revenue growth to 20% at the midpoint of today's updated guidance. The strengthening trajectory is just the beginning of BioMarin Pharmaceutical Inc.'s enhanced longer-term financial outlook supported by our larger, more diversified commercial portfolio. Since announcing our plans to acquire Amicus late last year, we have been preparing for rapid integration beginning on day one with the goal of increasing the peak potential of these newly added products. Following the recent close of the transaction, we initiated our targeted integration plan focused on leveraging BioMarin Pharmaceutical Inc.'s operating scale and capabilities to drive diagnosis and treatment rates for patients with Fabry disease and late-onset Pompe disease. Next quarter, we plan to share this roadmap as well as more detail on BioMarin Pharmaceutical Inc.'s growth acceleration with the addition of Galafold and Ponbility and Opholder to our commercial portfolio, DMX 200 for FSGS in phase three to our late-stage pipeline. Turning briefly to first quarter results and outlook for the remainder of this year and starting with enzyme therapies, I am pleased with the strong interest we are seeing from the PKU community following the recent Palynziq adolescent label expansion in the United States. With Cristin, who will discuss in more detail, we expect enzyme therapies will deliver robust growth in 2026, further supported by the addition of Galafold and Pongility and OptFolder to the portf...

The Amicus acquisition serves as a primary growth catalyst, accelerating 2026 revenue growth expectations to 20% at the midpoint via the addition of Galafold and Pombility/Opfolda. Voxzogo's performance is increasingly driven by early intervention strategies, with over half of new U.S. patient starts coming from the under-two-year-old cohort. Enzyme Therapies growth is supported by the Palynziq adolescent label expansion, which management views as a critical driver for long-term patient titration and maintenance. First-quarter revenue results were impacted by anticipated order timing dynamics and U.S. stocking normalization rather than shifts in underlying demand. Management is leveraging BioMarin's global scale to drive diagnosis rates for Fabry disease and switch rates for Pompe disease, targeting the large undiagnosed patient populations inherited from Amicus. Operational margins in Q1 were temporarily pressured by a $31 million manufacturing charge related to a Naglazyme process qualification, though commercial supply remains unaffected. Full-year 2026 revenue guidance was raised to $3.825B–$3.925B to reflect eight months of Amicus contribution, assuming high-teens to low-20s organic growth for the acquired assets. Profitability is expected to be heavily weighted toward the second half of 2026, with approximately two-thirds of annual EPS projected for Q3 and Q4 due to revenue timing. The Amicus acquisition is expected to be slightly dilutive for the full year 2026, but is projected to be accretive in the first twelve months after close and substantially accretive beginning in 2027 as cost synergies are realized. Management anticipates pivotal Phase 3 readouts for Voxzogo in hypochondroplasia and BMN-401 in ENPP1 deficiency in Q2 2026, which are critical for mid-term portfolio expansion. Guidance includes a modest allowance for potential disruption in the Middle East, reflecting management's monitoring of geopolitical uncertainties. A $31 million non-recurring charge was recorded in Q1 due to an unsuccessful manufacturing campaign to extend Naglazyme capability. The company is pursuing an exclusion order through the ITC to protect its intellectual property, with a decision on whether Ascendis' product infringes its patent expected around August 21. Integration risks are being managed through a 'day one' readiness plan, with a detailed roadmap for cost syn...