Welcome to our dedicated page for BioMarin Pharmaceuticals news (Ticker: BMRN), a resource for investors and traders seeking the latest updates and insights on BioMarin Pharmaceuticals stock.
BioMarin Pharmaceuticals Inc. (symbol: BMRN) is a global biotechnology firm dedicated to the development and commercialization of first-in-class and best-in-class therapeutics. Founded in 1997, the company's mission is to transform the lives of patients suffering from serious and life-threatening rare genetic diseases, particularly those affecting children. BioMarin targets rare conditions that are often inherited, difficult to diagnose, progressively debilitating, and lack effective treatment options.
BioMarin's diverse product portfolio includes independently marketed therapies such as Naglazyme, Vimizim, and Brineura, as well as Kuvan and Palynziq for treating phenylketonuria (PKU). The company also collaborates with Genzyme to market Aldurazyme. In 2021, BioMarin's Voxzogo (vosoritide) was approved for the treatment of achondroplasia, a rare genetic disorder affecting bone growth. Following this, in 2022, Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for hemophilia A, received approval in Europe and in the U.S. in 2023.
BioMarin places significant emphasis on research, development, manufacturing, and commercialization speed, aiming to deliver important therapies promptly. The company's capabilities have led to numerous transformational therapies addressing unmet medical needs, providing substantial benefits over existing treatments.
Notable recent developments include the expanded FDA approval for Voxzogo to increase linear growth in pediatric patients of all ages with achondroplasia with open epiphyses, and the substantial revenue growth contributed by global demand for Voxzogo. BioMarin also focuses on hypochondroplasia, launching a pivotal clinical trial program and presenting positive early results from a Phase 2 study.
Financially, BioMarin has demonstrated robust growth, with increased revenue driven by high demand for its products. The company has secured agreements with U.S. and European health authorities to expand patient access to Roctavian and other treatments. Strategic initiatives include corporate governance enhancements and collaborations with major investors to maximize long-term value creation.
For further details about ongoing clinical trials, product information, and patient support, visit www.biomarin.com.
BioMarin Pharmaceutical has received FDA approval for an increased maximum dose of 60 mg for its PKU treatment, Palynziq. Previously capped at 40 mg, this new dosage accommodates approximately 19% of participants from Phase 3 PRISM studies requiring higher doses. The label expansion includes long-term efficacy data showing sustained reductions in blood Phe levels, with 66% of participants achieving optimal levels after two years. BioMarin's ongoing commitment to the PKU community is evident as they continue to research and develop innovative therapies.
BioMarin Pharmaceutical has appointed Kevin Eggan, Ph.D., a MacArthur Fellowship winner, as the new Group Vice President for Research and Early Development as of October 5, 2020. With nearly two decades of expertise in rare neurological disorders, Dr. Eggan is expected to lead BioMarin's innovative research in genetic therapies. He aims to enhance drug discovery processes and ensure a robust pipeline of therapies for rare genetic diseases. Dr. Eggan joins from Harvard University, holding a strong publication record and numerous patents, and his arrival is set to bolster BioMarin's strategic vision.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has received Fast Track designation from the U.S. FDA for BMN 307, a gene therapy aimed at treating phenylketonuria (PKU). This designation will expedite the drug's development and review processes. The company's Phase 1/2 study, Phearless, is underway, evaluating the safety and efficacy of a single dose of BMN 307. With over 15 years in PKU research and two existing therapies, BioMarin aims to provide further treatment options. The Fast Track status may allow for Accelerated Approval and Priority Review.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that Brian Mueller, Executive Vice President and Chief Financial Officer, will participate in Morningstar's Management Behind the Moat Conference on September 30, 2020, at 1:00 PM ET. The conference will be accessible through a live audio webcast, which can be found on the company's investor relations website. An archived version will also be available for a limited time. BioMarin specializes in developing therapies for serious rare disorders and has six commercial products along with several candidates in its portfolio.
BioMarin Pharmaceutical (BMRN) has initiated the global PHEARLESS Phase 1/2 study by dosing the first participant with BMN 307, a gene therapy aimed at treating phenylketonuria (PKU). This investigational treatment seeks to normalize blood phenylalanine (Phe) levels by delivering a functional PAH gene. Both the FDA and EMA have granted Orphan Drug Designation to BMN 307, which represents a significant addition to BioMarin's PKU treatment portfolio. The study will assess the therapy's safety and efficacy, marking a crucial step in addressing the unmet needs of PKU patients.
BioMarin Pharmaceutical (NASDAQ: BMRN) will participate in four upcoming virtual investor conferences, as announced on September 15, 2020. Management will deliver presentations, available via audio webcast, on the company's innovative therapies for serious rare disorders. Interested parties can access the live webcast and an archived version through the company’s investor relations website. BioMarin's portfolio includes six commercialized products, with several candidates in clinical and pre-clinical stages. For more details, visit www.biomarin.com.
BioMarin Pharmaceutical has announced that data from its Phase 3 trial of vosoritide, aimed at treating achondroplasia in children aged 5-18, will be presented at the ASBMR Annual Meeting on September 12, 2020. This randomized, double-blind study involved 121 children and focused on growth velocity changes over one year. Vosoritide has received orphan drug designation from the FDA and EMA. The trial is part of a robust clinical program targeting a condition affecting 1 in 25,000 live births, with no current approved treatments in major markets.
BioMarin Pharmaceutical announced results from a Phase 3 trial of vosoritide, an investigational therapy for achondroplasia, published in The Lancet. Conducted on children aged 5-18, the trial showed that daily subcutaneous vosoritide significantly improved annualized growth velocity (AGV) by 1.57 cm/year compared to placebo (p < 0.0001). Additionally, height Z scores increased by +0.28 (p < 0.0001). The therapy was generally well tolerated with mild adverse effects. BioMarin plans to collaborate with regulatory authorities for potential marketing approvals, addressing a significant unmet medical need.
BioMarin Pharmaceutical (NASDAQ: BMRN) will participate in two virtual investor conferences, as announced on September 8, 2020. An audio webcast of the presentations will be available live, with access through their investor website. Archived versions of the remarks will also be provided for a limited time post-conference. BioMarin is a global biotechnology company focused on rare disorders, with six commercial products and several candidates in clinical development.
BioMarin Pharmaceutical (BMRN) announced the submission of a New Drug Application (NDA) to the FDA for vosoritide, a potential treatment for children with achondroplasia. This regulatory move follows the validation of the Marketing Authorization Application (MAA) by the EMA on Aug. 13, 2020. Vosoritide represents a significant milestone as the first pharmacological treatment option for this condition, which affects children characterized by disproportionate short stature. The drug is currently being tested in children under 18 with open growth plates, comprising 25% of achondroplasia cases.
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