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) delivers innovative therapies for rare genetic disorders through advanced biotechnology research. This news hub provides investors and healthcare professionals with timely updates on regulatory milestones, clinical trial progress, and strategic initiatives shaping the future of genetic medicine.
Access the most comprehensive collection of BioMarin news, including updates on enzyme replacement therapies, gene therapy advancements, and global commercialization efforts. Our curated feed ensures you stay informed about pipeline developments, partnership announcements, and financial performance without promotional bias.
Key updates cover FDA/EMA regulatory decisions, quarterly earnings insights, research collaborations, and manufacturing expansions. All content is verified through primary sources to maintain accuracy and compliance with financial disclosure standards.
Bookmark this page for streamlined tracking of BioMarin's progress in addressing unmet medical needs. Check regularly for objective reporting on therapeutic innovations impacting rare disease communities worldwide.
BioMarin (Nasdaq: BMRN) announced the FDA has accepted for Priority Review its supplemental biologics license application to expand PALYNZIQ (pegvaliase-pqpz) for adolescents aged 12–17 with phenylketonuria (PKU).
The sBLA is based on positive Phase 3 PEGASUS results showing statistically significant reductions in blood phenylalanine (Phe) versus diet alone. The FDA set a PDUFA target action date of Feb. 28, 2026. Data were presented at the 15th ICIEM in Kyoto, and BioMarin plans to seek a similar label expansion with the European Medicines Agency. PALYNZIQ is currently the first and only enzyme substitution therapy approved for adults with PKU.
BioMarin (NASDAQ: BMRN) reported Q3 2025 results and a corporate update on October 27, 2025. Total revenues were $776M (+4% vs Q3 2024) and YTD revenues reached $2,347M (+11% Y/Y). Year-to-date VOXZOGO revenue rose 24% Y/Y and Q3 VOXZOGO sales were $218M; PALYNZIQ grew 20% in Q3. GAAP net loss was $(31)M in Q3, driven primarily by a $221M acquired IPR&D charge from the Inozyme acquisition. Non-GAAP income fell to $22M in Q3 from $178M a year earlier. Cash and investments were about $1.99B and operating cash flow was $369M in Q3 ($728M YTD). The company reaffirmed VOXZOGO full-year guidance ($900M–$935M) and updated 2025 guidance, raising mid-point revenue while lowering Non-GAAP EPS and margin guidance to reflect the IPR&D charge. BioMarin plans to pursue options to divest ROCTAVIAN.
BioMarin (NASDAQ: BMRN) announced a conference call and webcast with Alexander Hardy, president and CEO, on Monday, October 27, 2025 at 4:30 p.m. ET to discuss third quarter 2025 financial results and provide a general business update.
Dial-in numbers are provided for U.S./Canada and international participants, and a live audio webcast will be available via the investor site at https://investors.biomarin.com/. A replay will be archived on the site for one week following the call.
BioMarin Pharmaceutical (Nasdaq: BMRN) presented new data from 14 studies at the ASBMR 2025 Annual Meeting, highlighting significant advances in bone health treatments. The key findings focus on VOXZOGO® (vosoritide) treatment outcomes in children with achondroplasia.
The Phase 2 CANOPY study demonstrated that children receiving VOXZOGO showed improved spinal morphology, with 57% experiencing reduced thoracolumbar kyphosis compared to 33% in the placebo group. Additionally, Phase 3 data revealed substantial growth benefits in post-puberty patients, with treated young men gaining 7.55 cm more and young women gaining 8.07 cm more in height compared to untreated individuals.
The company also presented research on ENPP1 deficiency, advancing BMN 401 as a potential first-in-disease enzyme therapy, with pivotal data expected in early 2026 and potential launch in 2027.
BioMarin (NASDAQ:BMRN) announced positive Phase 3 PEGASUS trial results for PALYNZIQ® in treating adolescents with phenylketonuria (PKU). The study demonstrated a 49.7% decrease in mean blood phenylalanine (Phe) levels compared to just 0.3% with diet alone.
Key achievements include: 51.6% of PALYNZIQ-treated patients achieved blood Phe levels ≤600 µmol/L, and 45.2% of participants achieved ≥50% reduction in blood Phe from baseline. The safety profile aligned with adult studies, though 5.6% experienced serious adverse events (anaphylaxis) leading to discontinuation.
BioMarin plans to submit these results to global health authorities in the second half of 2025 to expand PALYNZIQ's indication to include adolescent treatment.
BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its participation in three major healthcare investor conferences in September 2025:
- Cantor Global Healthcare Conference on September 3 at 8:00 AM EST
- Wells Fargo Healthcare Conference on September 4 at 8:45 AM EST
- Morgan Stanley Global Healthcare Conference on September 9 at 11:30 AM EST
The company will provide live audio webcasts of the presentations through their investor relations website, with archived versions available for limited time after each event.
BioMarin (NASDAQ:BMRN) reported strong Q2 2025 results with total revenues of $825 million, up 16% year-over-year. The company achieved GAAP EPS of $1.23 (+124% Y/Y) and Non-GAAP EPS of $1.44 (+50% Y/Y).
Key highlights include VOXZOGO revenue growth of 20% and Enzyme Therapies revenue growth of 15%. The company completed the acquisition of Inozyme in July 2025, adding BMN 401 to its portfolio. Additionally, BMN 333, their long-acting CNP for achondroplasia, showed promising results in healthy volunteer studies.
Based on strong performance, BioMarin raised its full-year 2025 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS. The company ended Q2 with $1.9 billion in cash and investments.
BioMarin Pharmaceutical (Nasdaq: BMRN) has appointed Ian T. Clark to its Board of Directors, effective August 1, 2025. Clark, the former CEO of Genentech (2010-2016), brings significant industry experience, having overseen the launch of 15 new drugs during his tenure. Prior to Genentech, he held senior positions at major pharmaceutical companies including Novartis, Sanofi, Ivax, and G.D. Searle.
Clark currently serves on the boards of several biotech companies including Olema Oncology, Takeda Pharmaceutical, and Guardant Health. He is also an advisor to KKR & Co. Inc. His appointment brings valuable expertise in drug development and commercialization to BioMarin's board.
BioMarin Pharmaceutical (NASDAQ: BMRN) has scheduled its second quarter 2025 financial results conference call and webcast for Monday, August 4, 2025, at 4:30 p.m. ET. The company's President and CEO, Alexander Hardy, will lead the discussion of Q2 2025 financial performance and provide business updates.
Investors and interested parties can access the live audio webcast through BioMarin's investor relations website. A replay will be available for one week after the call. The company has provided both domestic and international dial-in numbers for telephone access to the conference call.
BioMarin Pharmaceutical (Nasdaq: BMRN) has completed its acquisition of Inozyme Pharma (Nasdaq: INZY) in an all-cash transaction valued at approximately $270 million, or $4.00 per share. The tender offer, which expired on June 30, 2025, resulted in approximately 70% of Inozyme shares being validly tendered.
The strategic acquisition strengthens BioMarin's enzyme therapies portfolio by adding INZ-701, a late-stage enzyme replacement therapy. This investigational treatment targets ENPP1 Deficiency, a rare genetic condition affecting blood vessels, soft tissues, and bones. INZ-701 is designed to restore pyrophosphate and adenosine levels and has potential applications in additional indications including ABCC6 Deficiency and calciphylaxis.
Following the merger completion, Inozyme shares have ceased trading on the Nasdaq Global Select Market and will be delisted. The Boston-based Inozyme, with approximately 50 employees, brings expertise in the PPi-Adenosine Pathway research and development to BioMarin's established portfolio of eight commercial therapies.
FAQ
What is the current stock price of Biomarin Pharmaceutical (BMRN)?
What is the market cap of Biomarin Pharmaceutical (BMRN)?
