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 (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.
BioMarin (NASDAQ:BMRN) presented compelling five-year Phase 3 results for ROCTAVIAN®, their gene therapy treatment for severe hemophilia A, at the ISTH 2025 Congress. The GENEr8-1 trial demonstrated sustained efficacy with mean Factor VIII activity remaining in the mild hemophilia range (24.0 IU/dL one-stage assay), and 73.5% of participants maintaining FVIII levels in the mild-to-normal range.
Key outcomes after five years include: 81.3% of participants remaining off prophylaxis, mean annualized bleeding rate of just 0.6 bleeds/year, and 77.8% of participants experiencing zero treated bleeds during year five. The study confirmed consistent safety with no new signals, no FVIII inhibitors, no thromboembolic events, and no treatment-related malignancies.
Treatment demonstrated significant reduction in tibial bowing compared to placebo in children with achondroplasia, with sustained improvement over several years. Modeling indicated potential final height increases of 21.7 cm in girls and 26.4 cm in boys compared to untreated children when treated early and continuously.
A large retrospective study of over 600 patients with hypochondroplasia revealed higher rates of comorbidities, surgeries, and doctor visits compared to those without the condition. Additionally, early data from a Phase 2 trial showed promising increases in annualized growth velocity for girls with Turner syndrome, even in those who previously had suboptimal results with human growth hormone.
BioMarin has completed enrollment in its pivotal Phase 3 study for VOXZOGO in hypochondroplasia, with topline data expected in 2026 and potential launch in 2027.
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.