Welcome to our dedicated page for Biomarin Pharmaceutical news (Ticker: BMRN), a resource for investors and traders seeking the latest updates and insights on Biomarin Pharmaceutical stock.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is a global rare disease biotechnology company focused on medicines for genetically defined conditions. This news page aggregates company announcements, press releases and event updates so readers can follow how BioMarin’s strategy, pipeline and commercial portfolio evolve over time.
Recent communications from BioMarin highlight its emphasis on Enzyme Therapies and Skeletal Conditions business units, as well as its work with ROCTAVIAN, a gene therapy for hemophilia A. The company issues news on topics such as financial results, long-term guidance, corporate strategy, business development transactions, clinical data presentations and regulatory milestones for its therapies.
Examples of news themes include updates on VOXZOGO in achondroplasia and other skeletal conditions, progress with PALYNZIQ in phenylketonuria, development of BMN 401 for ENPP1 deficiency and other pipeline programs in rare genetic diseases. BioMarin also reports on strategic partnerships, such as digital and data collaborations, and on planned acquisitions intended to expand and diversify its rare disease portfolio.
Investors and followers of BMRN can use this page to review earnings announcements, conference presentations, investor day materials and product-specific updates released through newswires. By checking this feed regularly, readers can see how BioMarin describes its growth priorities, clinical milestones and capital allocation decisions across its rare disease franchises.
BioMarin Pharmaceutical (NASDAQ: BMRN) has scheduled its first quarter 2025 financial results conference call and webcast for Thursday, May 1, 2025, at 4:30 p.m. ET. The event will be hosted by Alexander Hardy, President and Chief Executive Officer, who will discuss Q1 2025 financial performance and provide a business update.
Participants can join via phone using U.S./Canada dial-in (888-596-4144) or International dial-in (646-968-2525) with Conference ID 4327591. A live audio webcast will be available through BioMarin's investor website. A replay will be accessible for one week following the call.
BioMarin Pharmaceutical (Nasdaq: BMRN) announced positive Phase 3 PEGASUS trial results for PALYNZIQ® (pegvaliase-pqpz) in treating adolescents with phenylketonuria (PKU). The study met its primary efficacy endpoint, showing statistically significant reduction in blood Phe levels among patients aged 12-17 compared to diet alone.
The safety profile aligned with known results, and PALYNZIQ remains the first and only enzyme therapy approved for adult PKU treatment. BioMarin plans to present detailed findings at an upcoming medical meeting and will submit to global health authorities later this year to expand PALYNZIQ's label for adolescent use.
BioMarin (BMRN) presented new data for two key medicines at the 2025 ACMG Annual Meeting. A Japanese study of VOXZOGO in children under 3 with achondroplasia showed strong treatment adherence with no reported treatment-related adverse events across 63 children followed for up to 23.7 months.
The PALYNZIQ OPAL study demonstrated significant blood Phe level reduction from 1029 μmol/L at baseline to 293 μmol/L at week 96, representing a 67.8% decrease. Quality of life improvements were observed through PKU-QOL and PKU-SSIS measurements. Additional analyses from the Phase 3 PRISM trial showed that maintaining lower blood Phe levels led to improvements in attention and mood.
The ATLAS study revealed an increased proportion of PKU patients achieving target blood Phe levels ≤360 μmol/L across 19 U.S. clinics, with Phase 3 results in adolescents expected later this year.
BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its upcoming participation at the TD Cowen 45th Annual Healthcare Conference in Boston, MA. The company's management will deliver a presentation on Tuesday, March 4, 2025, at 6:50 AM PT / 9:50 AM ET.
The presentation will be accessible through a live audio webcast via the company's investor relations website at investors.biomarin.com. For those unable to attend live, an archived version of the presentation will be made available on BioMarin's website for a time after the conference.
BioMarin Pharmaceutical (Nasdaq: BMRN) has appointed Timothy P. Walbert to its Board of Directors, effective Feb. 24, 2025. Walbert, the former chairman, president and CEO of Horizon Therapeutics until its acquisition by Amgen in 2023, brings over 30 years of pharmaceutical industry experience.
Walbert's extensive background includes leadership roles at IDM Pharma (acquired by Takeda), NeoPharm, Abbott (now AbbVie), Pharmacia, Merck, Pfizer, and Wyeth Ayerst. He currently serves as senior advisor at Amgen and holds board positions at Sagimet Biosciences, Mirum Pharmaceuticals, and Century Therapeutics.
The appointment aligns with BioMarin's new strategy focused on innovation, growth, and value creation, with CEO Alexander Hardy highlighting Walbert's operational expertise and patient-centered approach in commercializing medicines.
BioMarin Pharmaceutical (NASDAQ: BMRN) reported robust financial results for Q4 and FY 2024, with total revenues of $747 million for Q4 (+16% Y/Y) and $2.85 billion for the full year (+18% Y/Y). The growth was driven by strong contributions from VOXZOGO and Enzyme Therapies. GAAP net income for Q4 increased by $105 million to $125 million, while non-GAAP income rose by $85 million to $180 million. The company projects double-digit revenue and profitability growth in 2025, with total revenues expected to grow by 10% Y/Y.
Key developments include the advancement of pipeline candidates such as BMN 351 for Duchenne Muscular Dystrophy and BMN 333 for skeletal conditions, with significant clinical data expected in 2025. VOXZOGO saw a 42% increase in Q4 and 56% in FY 2024, and is on track to be available in over 60 countries by 2027.
BioMarin's full-year GAAP operating margin expanded by 9.3 percentage points to 17.0%, with a GAAP diluted EPS of $2.21 (+154% Y/Y). The company expects a non-GAAP operating margin of 40% by 2026. Operating cash flows for FY 2024 totaled $573 million, a 260% increase Y/Y, with total cash and investments at $1.7 billion. BioMarin targets over $1.25 billion in operating cash flows annually starting in 2027.
BioMarin Pharmaceutical (NASDAQ: BMRN) has scheduled its fourth quarter and full-year 2024 financial results conference call and webcast for Wednesday, February 19, 2025, at 4:30 p.m. ET. The event will be hosted by Alexander Hardy, President and Chief Executive Officer, who will discuss financial results and provide a general business update.
Participants can join via phone using U.S./Canada dial-in (800-715-9871) or International dial-in (646-307-1963) with Conference ID 1878833. A live audio webcast will be available through BioMarin's investor website. A replay will be accessible for one week following the call, with U.S./Canada replay number (800-770-2030) and International replay number (609-800-9909), Playback ID 1878833.
BioMarin Pharmaceutical (Nasdaq: BMRN) has initiated legal action against Ascendis Pharma A/S in the Unified Patent Court (UPC) in Munich, Germany, for alleged infringement of European patent EP 3 175 863 B1. The patent, which covers long-acting variants of C-Type Natriuretic Peptide (CNP), was confirmed by the European Patent Office's Opposition Division in 2024.
The legal action targets Ascendis' TransCon CNP investigational product and its development program in Germany and Europe. A decision is expected within 12-15 months. The UPC, operational since June 2023, has jurisdiction over European patents across 18 European member states that have ratified the Unitary Patents.
BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, California. Alexander Hardy, the company's President and Chief Executive Officer, will deliver a presentation on Monday, January 13, 2024, at 9:00 am PT / 12:00 pm ET.
The presentation will be accessible through a live audio webcast on the company's investor relations website. For those unable to attend or watch live, an archived version of the presentation will be available for a time through BioMarin's website.
BioMarin Pharmaceutical (Nasdaq: BMRN) presented positive real-world evidence for VOXZOGO in treating children with achondroplasia at the ESPE Meeting 2024. Data from the European CrescNet registry, involving 452 children across 30 centers, showed significant height improvements. After 12 months of treatment, 143 participants averaged a 6.36 cm height increase, while 73 participants treated for 24 months showed an 11.86 cm increase. A separate French study of 17 children above age 5 demonstrated an average 8.76 cm height increase over 18 months, with no treatment discontinuations. The company has collected over 6,000 patient-years of safety data through their CANOPY clinical program.
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