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.
The FDA has approved BioMarin's supplemental Biologics License Application (sBLA) for BRINEURA® (cerliponase alfa) to treat children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), including those under 3 years, regardless of symptom presence. Previously, BRINEURA was only indicated for symptomatic children aged 3 and above. The approval is based on data from Study 190-203, a Phase 2 trial that showed BRINEURA slows motor function decline and delays disease onset in children aged 1-6, including those under 3. In this study, none of the treated children under 3 experienced a significant motor decline, compared to 61% of untreated children in a natural history cohort. The safety profile for children under 3 is consistent with the known safety profile of the drug. This expanded indication emphasizes the importance of early diagnosis and treatment for CLN2 disease.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that it will host a conference call and webcast on Monday, August 5, 2024, at 4:30 p.m. ET to discuss its second quarter 2024 financial results and provide a general business update. The call will be hosted by Alexander Hardy, President and Chief Executive Officer of BioMarin.
Participants can join the call using the following details:
- U.S. / Canada Dial-in: 888-596-4144
- International Dial-in: 646-968-2525
- Conference Call ID: 1816377
BioMarin announced new data from its Phase 2 111-205 study showing that children with achondroplasia treated with VOXZOGO® exhibited significant increases in bone length while maintaining bone strength over five years. The results will be presented at the International Conference on Children's Bone Health in June 2024. Additional Phase 3 data reveal VOXZOGO's positive impacts on proportionality and health-related quality of life. Separately, research on hypochondroplasia highlights higher comorbidity rates, underscoring unmet medical needs. BioMarin is advancing clinical trials for hypochondroplasia, idiopathic short stature, and other growth conditions.
BioMarin presented four-year data from its Phase 3 study on ROCTAVIAN® at the ISTH 2024 Congress, showing long-term safety and efficacy in treating severe hemophilia A. The study revealed that 73.6% of patients had zero treated bleeds in year four, with stable FVIII levels. Mean FVIII activity was 27.1 and 16.1 IU/dL by OSA and CSA assays, respectively. Additionally, there was a notable improvement in health-related quality of life (HRQoL), with a 6.2 point increase in Haemo-QOL-A Total Score. Phase 2 results also showed seven-year bleed control for a majority of patients.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will be presenting at the BofA Securities 2024 Healthcare Conference on May 15, 2024, in Las Vegas, NV. Alexander Hardy, President, and CEO of BioMarin, will be presenting at 10:00am PT / 1:00pm ET. The presentation will be available via live audio webcast and archived on the company's website.
BioMarin Pharmaceutical Inc. presented positive new data at the 2024 Pediatric Endocrine Society Annual Meeting, highlighting the safety and efficacy of VOXZOGO® (vosoritide) in children with achondroplasia and other growth-related conditions. Results from multiple studies showed significant improvements in annualized growth velocity and height standard deviation, particularly in children with Noonan syndrome, ACAN deficiency, and NPR2 mutations. Data also demonstrated VOXZOGO's potential to positively impact growth beyond achondroplasia. Clinical trials for growth-related conditions are ongoing, with promising results for children with achondroplasia who initiated treatment during adolescence.
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