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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 a conference call scheduled for February 25, 2021, at 4:30 p.m. ET. CEO Jean-Jacques Bienaimé will discuss the company's fourth quarter and full year 2020 financial results alongside a business update. The call can be accessed via a live audio webcast on the BioMarin website. For U.S. participants, the dial-in number is (866) 502-9859, while international participants can dial (574) 990-1362, using Conference ID 6488682.
BioMarin Pharmaceutical (BMRN) announced positive topline results from its Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, a gene therapy for severe hemophilia A. The trial included 134 participants and found an 84% reduction in Annualized Bleeding Rate (ABR) after one year, with 80% of participants being bleed-free by week five. Factor VIII infusions also decreased by 99%. The FDA granted Breakthrough Therapy Designation, and BioMarin plans to submit data for marketing approval. The study represents a significant step toward a new treatment paradigm for hemophilia A.
BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its participation in two upcoming virtual investor conferences. An audio webcast of these presentations will be available live, with access provided through the company's investor relations website. Additionally, an archived version will be accessible for a limited time post-conference. BioMarin specializes in innovative therapies for serious rare disorders, boasting a portfolio of six commercialized products along with several candidates in clinical stages.
BioMarin Pharmaceutical announced positive results from the Phase 3 extension study of vosoritide, a treatment for children with achondroplasia. Following two years of treatment, children showed maintained growth acceleration with a cumulative height gain of 3.52 cm compared to untreated subjects. The treatment was well tolerated, with 93% of participants remaining on it. Regulatory applications for vosoritide are under review by the FDA and EMA, with potential approval dates in August and the second half of 2021, respectively. This could mark the first therapy for achondroplasia in these regions.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced its participation in two upcoming virtual investor conferences. An audio webcast of the presentations will be available for live access and later archived on the company's website. BioMarin focuses on developing innovative therapies for life-threatening rare disorders, boasting a portfolio that includes six commercialized products along with several clinical and pre-clinical candidates. For further details, please visit www.biomarin.com.
BioMarin Pharmaceutical (BMRN) announced a preclinical collaboration with Deep Genomics on November 17, 2020, to enhance drug discovery using AI technology. The partnership focuses on identifying oligonucleotide drug candidates for four rare diseases with significant unmet needs. Financial terms remain undisclosed, but Deep Genomics will receive an upfront payment and potential development milestones. This collaboration aims to leverage AI for faster identification and validation of therapeutic candidates, aiming to expedite BioMarin's development pipeline in rare disease treatments.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced its participation in three upcoming virtual investor conferences on November 12, 2020. Management will be presenting, and an audio webcast will be available live on their website. Archived versions of the presentations will be accessible for a limited time post-conference. BioMarin focuses on developing innovative therapies for serious rare disorders and has a strong portfolio with six commercialized products and ongoing clinical candidates. Further details can be found on their official website.
BioMarin Pharmaceutical (NASDAQ:BMRN) has announced the expansion of its clinical program for vosoritide with two new Phase 2 studies targeting children with achondroplasia and other genetic forms of short stature. The first study will assess the safety of vosoritide in infants at risk of life-threatening foramen magnum compression. The second, an investigator-initiated study by Children's National Hospital, will explore vosoritide’s effects on growth in selected genetic short stature patients. Both studies aim to enroll a significant number of participants, aiming to enhance treatment outcomes.
BioMarin Pharmaceutical reported Q3 2020 financial results with total revenues of $476.8 million, up by 3% from Q3 2019. Net product revenues increased by 2% to $460.7 million. The company achieved a substantial GAAP net income of $784.8 million, largely due to a $800.8 million tax benefit related to IP transfer. However, revenues from products like Vimizim and Naglazyme declined. Despite COVID-19 challenges affecting revenues, BioMarin remains optimistic about upcoming regulatory filings for its gene therapies, valoctocogene roxaparvovec and vosoritide. Cash reserves stood at $1.77 billion.
BioMarin Pharmaceutical (NASDAQ: BMRN) announced that its CEO, Jean-Jacques Bienaimé, and CFO, Brian Mueller, will participate in the Credit Suisse 29th Annual Virtual Healthcare Conference on November 10, 2020, at 12:30 PM ET. An audio webcast will be available live and can be accessed here. An archived version will also be accessible on the company's website afterward. BioMarin develops innovative therapies for serious rare disorders and has six commercialized products along with several candidates in the pipeline.
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