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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. (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.
BioMarin Pharmaceutical (BMRN) reported strong financial results for Q2 2021, with total revenues reaching $501.7 million, up 17% from $429.5 million in Q2 2020. The net product revenues rose 19%, driven by Vimizim and Naglazyme sales. Notably, GAAP net income improved to $12.9 million from a loss of $29.2 million year-over-year. BioMarin also announced the positive CHMP opinion for VOXZOGO, targeting regulatory approval in Europe and the U.S. in late 2021. Full-year revenue guidance was revised upward to between $1.790 billion and $1.880 billion.
BioMarin Pharmaceutical announced new data for valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, at the ISTH 2021 Virtual Congress. The Phase 1/2 study showed significant long-term benefits, with 95% reduction in annualized bleed rates (ABR) in the high-dose cohort after five years. All participants in this cohort remained off prophylactic Factor VIII treatment. Factor VIII usage dropped by 96%, with favorable safety profiles reported, sustaining efficacy over time. Regulatory submissions for accelerated assessment are underway in both the EU and the US.
BioMarin announced promising results from the GENEr8-1 pivotal study of valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, during the ISTH 2021 Congress. In this largest Phase 3 study involving 134 participants, over 90% achieved an annualized bleeding rate (ABR) of zero after treatment. The therapy also reduced Factor VIII utilization by 99%. The data support the therapy's potential to transform hemophilia A treatment, with significant increases in endogenous Factor VIII levels observed. The company plans to submit regulatory applications for marketing approval in 2022.
BioMarin Pharmaceutical announced the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for valoctocogene roxaparvovec, a gene therapy for severe hemophilia A. This initiates the review process, with a CHMP opinion expected in the first half of 2022. The MAA includes data from the Phase 3 GENEr8-1 study involving 134 participants. The EMA previously granted an accelerated assessment for this application, highlighting its public health significance. BioMarin plans to resubmit its Biologics License Application (BLA) in the U.S. in Q2 2022.
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.
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