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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 (BMRN) has announced promising long-term data on VOXZOGO® (vosoritide) for treating achondroplasia. Recent studies show sustained growth velocity improvements over seven years in children treated with VOXZOGO, with annualized growth velocities (AGVs) surpassing untreated peers by 1.90 cm/year for boys and 1.36 cm/year for girls. The drug maintained a favorable safety profile with no new adverse effects. VOXZOGO is the first approved therapy for this condition and is currently under review for younger patients. The latest findings will be presented at the ACMG Annual Meeting in Salt Lake City.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that the FDA has accepted its supplemental New Drug Application (sNDA) to expand the use of VOXZOGO (vosoritide) for treating children under 5 with achondroplasia. This update follows positive results from a global Phase 2 study showing similar safety and efficacy in younger patients compared to older ones. The FDA's PDUFA action date is set for October 21, 2023. Currently, there are no approved pharmacological treatments for this age group in the U.S., potentially expanding access to over 1,000 additional children.
BioMarin Pharmaceutical Inc. announced that the FDA has extended the PDUFA target action date for the Biologics License Application of its gene therapy, ROCTAVIAN (valoctocogene roxaparvovec), for adults with severe hemophilia A to June 30, 2023. This extension follows the submission of a three-year data analysis from the ongoing Phase 3 GENEr8-1 study, which involved 134 participants, marking it as the largest gene therapy trial for hemophilia to date. BioMarin has received multiple designations for this therapy, including Regenerative Medicine Advanced Therapy and Orphan Drug status, reflecting its potential to address significant unmet medical needs.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced participation in three investor conferences in March 2023. Management will be presenting at the Cowen Healthcare Conference on March 8 at 10:30 am ET, followed by the Barclays Global Healthcare Conference on March 15 at 9:30 am ET. Additionally, they will speak at the Jefferies Biotech on the Bay Summit on March 16 in Miami. The events will be webcast live, and replays will be available for a limited time on BioMarin's website.
BioMarin, founded in 1997, focuses on developing therapies for rare genetic disorders, with a strong pipeline of treatments.
BioMarin Pharmaceutical reported record 2022 total revenues of $2.1 billion, a 14% increase from 2021. The fourth-quarter revenues reached $537.5 million, up 19%. Expecting 15% growth in overall revenues and 30% growth in net income for 2023, the company is focused on the launches of VOXZOGO and ROCTAVIAN. VOXZOGO generated $169.1 million in 2022, while ROCTAVIAN has a projected revenue of $100 to $200 million for 2023. Despite a net loss of $0.2 million in Q4 2022, BioMarin anticipates maintaining operational excellence.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will participate virtually in the SVB Securities Global Biopharma Conference on February 16, 2023, at 11:20 a.m. ET. Company leaders, Jean-Jacques Bienaimé and Brian Mueller, will represent the firm. Investors can access the live webcast at BioMarin's investor website. An archived version will also be available for a limited time post-conference. BioMarin, founded in 1997, focuses on genetic therapies and has developed eight commercial therapies for rare genetic disorders to meet significant medical needs. For more information, visit www.biomarin.com.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will hold a conference call on February 27 at 4:30 p.m. ET to discuss its fourth quarter and full-year 2022 financial results. CEO Jean-Jacques Bienaimé will lead the call, providing a general business update. Investors can dial in using the U.S. number 800-831-4163 or international number 213-992-4616. A replay will be available after the call. BioMarin, founded in 1997, focuses on developing targeted therapies for rare genetic disorders, with a strong pipeline of commercial and clinical candidates addressing significant unmet medical needs.
BioMarin Pharmaceutical announced promising results from the Phase 3 GENEr8-1 study of ROCTAVIAN, a one-time gene therapy for severe hemophilia A, with data covering over three years. Key findings include an 80% reduction in annualized bleed rates and a 94% decrease in Factor VIII usage. Notably, 92% of patients remained off prophylactic treatment by Year 3. The company signed its first outcomes-based agreement in Germany, with more expected soon. Additionally, the FDA completed a pre-license inspection of BioMarin's manufacturing facility, further supporting ROCTAVIAN's regulatory review.
BioMarin Pharmaceutical (NASDAQ: BMRN) will present at the 41st Annual J.P. Morgan Healthcare Conference on January 9, 2023, at 10:30 am PT in San Francisco, California. CEO Jean-Jacques Bienaimé will lead the presentation. Interested investors can access the live webcast on www.biomarin.com. The replay will be available for a week post-event.
BioMarin has submitted a Supplemental New Drug Application (sNDA) to the FDA to expand the use of VOXZOGO for treating children with achondroplasia under age 5. The submission follows positive results from a Phase 2 study showing increased growth velocity across all age cohorts. The company aims to help over 1,000 eligible children in the U.S. and Europe. Regulatory decisions are anticipated in the second half of 2023. VOXZOGO is already approved for older children with the same condition and aims to support growth in those with open growth plates.
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