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 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 Inc. (BMRN) will host a conference call and webcast on July 28, 2021, at 4:30 p.m. ET to discuss its second quarter financial results. The call will be led by Jean-Jacques Bienaimé, Chairman and CEO. Investors can access the live audio via BioMarin's investor website, with a replay available for one week post-call. As a biotechnology company, BioMarin focuses on innovative therapies for serious genetic diseases, holding a portfolio of six commercial products and multiple candidates under development.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced 12 presentations related to valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, at the ISTH 2021 Virtual Congress from July 17-21, 2021. This includes significant findings from the Phase 3 GENEr8-1 trial, which is the largest gene therapy trial for hemophilia A, showing prolonged hemostatic efficacy. BioMarin resubmitted a Marketing Authorization Application to the EMA on June 25, 2021, with a CHMP opinion expected in H1 2022, and plans for a BLA resubmission to FDA in Q2 2022.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has resubmitted a Marketing Authorization Application (MAA) for its gene therapy, valoctocogene roxaparvovec, targeting adults with severe hemophilia A. The European Medicines Agency (EMA) granted the request for accelerated assessment, potentially expediting the review process. The application includes data from the 134 participants in the Phase 3 GENEr8-1 study. A CHMP opinion is expected in H1 2022, while a Biologics License Application (BLA) resubmission is planned for Q2 2022 in the U.S., contingent on favorable results.
BioMarin Pharmaceutical has received a positive opinion from the CHMP for vosoritide, a treatment for achondroplasia in children aged 2 until growth plates close. A final approval from the European Commission is anticipated in Q3 2021. Over 11,000 children in Europe, the Middle East, and Africa may benefit from this first pharmacological option. The French health authority has granted temporary authorization for immediate access to vosoritide. The treatment has shown promising safety and efficacy outcomes in clinical studies, with no serious adverse events linked to the drug.
BioMarin Pharmaceutical (NASDAQ: BMRN) will present at the BofA Securities 2021 Napa Biopharma Conference on June 16, 2021, at 3:30 PM ET. An audio webcast of the presentation will be available live, with an archived version accessible on the company's website for a limited time afterwards. BioMarin focuses on developing therapies for rare disorders and has a diverse portfolio of six commercialized products alongside several clinical candidates. For more details, visit www.biomarin.com.
BioMarin Pharmaceutical announced its participation in two virtual investor conferences on May 27, 2021. An audio webcast of the presentations will be available live through the company's investor relations website. Additionally, an archived version of the remarks will be accessible for a limited time after the conferences. BioMarin, a global biotechnology company, specializes in innovative therapies for serious rare disorders and has a portfolio of six commercial products alongside multiple clinical candidates. For more details, visit www.biomarin.com.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced that the European Medicines Agency (EMA) has granted accelerated assessment for valoctocogene roxaparvovec, aimed at treating adults with severe hemophilia A. This expedited review process is expected to shorten the evaluation timeline, with a Committee for Medicinal Products for Human Use (CHMP) opinion anticipated in the first half of 2022. BioMarin plans to submit a Marketing Authorization Application (MAA) in June 2021, including 52 weeks of Phase 3 GENEr8-1 trial data.
BioMarin Pharmaceutical has shared a significant update regarding its investigational gene therapy valoctocogene roxaparvovec for severe hemophilia A. The latest data shows sustained treatment benefits over five years, with participants remaining off prophylactic Factor VIII treatment. The mean annualized bleed rate in year five was 0.7, representing a 95% reduction from baseline. The company plans to submit a Marketing Authorization Application in Europe in June 2021 and a Biologics License Application in the U.S. in Q2 2022, contingent on favorable study results.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced participation in two virtual conferences, with live audio webcasts available for investors. The webcasts can be accessed at biomarin.com. An archived version will also be accessible post-conference. BioMarin focuses on developing therapies for rare disorders, boasting a portfolio of six commercial products and various clinical candidates. For more details, visit their website.
BioMarin Pharmaceutical announced three oral and three poster presentations for its investigational gene therapy, valoctocogene roxaparvovec, at the ASGCT Virtual 2021 Annual Meeting. These presentations enhance the understanding of the therapy's potential for treating adults with severe hemophilia A. The company plans to submit a Marketing Authorization Application in Europe and a Biologics License Application in the U.S. by mid-2022, contingent on favorable study results. BioMarin also maintains a strong focus on manufacturing capabilities, aiming to produce 10,000 doses per year.
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