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 has received accelerated FDA approval for VOXZOGO™ (vosoritide), aimed at promoting linear growth in children aged five and older with achondroplasia. This approval is based on significant improvements in annualized growth velocity (AGV) observed in clinical trials, where Voxzogo showed an AGV increase of 1.57 cm/year. The therapy, a first-of-its-kind treatment targeting the root cause of achondroplasia, is expected to be available in the US by mid-December. The FDA also issued a Rare Pediatric Disease Priority Review Voucher to BioMarin.
Entos Pharmaceuticals and BioMarin Pharmaceutical have entered an agreement to use the Fusogenix platform to formulate product candidates for genetic diseases. This collaboration aims to leverage Entos' innovative nucleic acid delivery technology to enhance BioMarin's gene therapy pipeline. Fusogenix employs proprietary lipid formulations for effective delivery of mRNA and DNA. BioMarin will conduct preclinical studies on these formulations to evaluate their therapeutic potential. This partnership underscores BioMarin's commitment to advancing transformative therapies for patients with rare genetic conditions.
BioMarin Pharmaceutical reported third-quarter 2021 revenues of $408.7 million, down 14% from $476.8 million in Q3 2020. Net product revenues fell 12% to $369.5 million, with Kuvan revenues plummeting 45%. GAAP net loss reached $36.5 million, contrasting sharply with a $784.8 million gain in Q3 2020, primarily due to a one-time tax benefit. However, the company anticipates revenue growth in Q4 2021 and a strong 2022, driven by Voxzogo's recent EU approval and upcoming U.S. application. Full-year revenue guidance was revised to $1.820-$1.880 billion.
Summary not available.
BioMarin Pharmaceutical (NASDAQ: BMRN) will participate in four virtual investor conferences, as announced on October 25, 2021. Live audio webcasts of the presentations will be accessible via the company's investor relations website. Archived recordings will also be available for a limited time post-conference. BioMarin focuses on developing therapies for rare genetic diseases, with a range of commercial and clinical product candidates. For more details, visit www.biomarin.com.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced a conference call on October 27 at 4:30 p.m. ET to discuss its third quarter 2021 financial results and provide a general business update. The call will be hosted by Jean-Jacques Bienaimé, the company's CEO. Investors can join via a U.S. or international dial-in number or through a live audio webcast on the BioMarin website. A replay will be available for one week after the call.
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced the hiring of Harold S. Bernstein as Senior Vice President and Chief Medical Officer, and Ganesh Vedantham as Senior Vice President of Technical Development. Dr. Bernstein, with over 30 years of experience, will lead clinical development and succeed Geoff Nichol. Dr. Vedantham, a leader in CMC functions, will replace Victoria Sluzky, who is retiring after 19 years. CEO Jean-Jacques Bienaimé emphasized their potential to enhance the company’s pipeline for rare genetic diseases.
BioMarin Pharmaceutical announced that the FDA has placed a clinical hold on the BMN 307 Phearless Phase 1/2 study, which evaluates an investigational gene therapy for phenylketonuria (PKU). The hold is due to safety concerns identified in a pre-clinical study, revealing that 6 out of 7 mice given the highest dose developed tumors linked to AAV vector integration. BioMarin will pause further participant enrollment while investigating these findings, although no tumors have been reported in humans. They are committed to monitoring and evaluating the health of participants who received lower doses.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that the European Commission has granted marketing authorization for VOXZOGO (vosoritide), the first approved medicine for treating achondroplasia in children aged 2 until their growth plates close. This approval is based on clinical data showing sustained growth rates without increased bone age. Over 11,000 children across Europe, the Middle East, and Africa are affected by achondroplasia, and vosoritide aims to provide a targeted therapy option to improve height and quality of life. The FDA is also reviewing its NDA for vosoritide, with a PDUFA target date of November 20, 2021.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced participation in three virtual investor conferences on August 25, 2021. Live audio webcasts will be accessible on the company's investor relations page. Archived presentations will also be available for a limited period. BioMarin specializes in therapies for serious rare disorders, with six commercial products and various clinical candidates in its portfolio. For more details, visit www.biomarin.com.