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BioMarin Pharmaceutical Inc. develops and commercializes medicines for rare genetic diseases, with recurring news centered on commercial portfolio performance, regulatory approvals, clinical data and business development. Company updates commonly reference therapies for achondroplasia, phenylketonuria, hemophilia, mucopolysaccharidosis and lysosomal storage diseases, including VOXZOGO, PALYNZIQ, GALAFOLD and POMBILITI + OPFOLDA.
BioMarin news also covers financial results, guidance, acquisition integration, debt financing and governance changes. Clinical and regulatory announcements focus on genetically defined conditions, skeletal disorders, enzyme-based therapies and investigational rare-disease programs such as DMX-200 for focal segmental glomerulosclerosis.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that it will host a conference call and webcast on Monday, August 5, 2024, at 4:30 p.m. ET to discuss its second quarter 2024 financial results and provide a general business update. The call will be hosted by Alexander Hardy, President and Chief Executive Officer of BioMarin.
Participants can join the call using the following details:
- U.S. / Canada Dial-in: 888-596-4144
- International Dial-in: 646-968-2525
- Conference Call ID: 1816377
BioMarin announced new data from its Phase 2 111-205 study showing that children with achondroplasia treated with VOXZOGO® exhibited significant increases in bone length while maintaining bone strength over five years. The results will be presented at the International Conference on Children's Bone Health in June 2024. Additional Phase 3 data reveal VOXZOGO's positive impacts on proportionality and health-related quality of life. Separately, research on hypochondroplasia highlights higher comorbidity rates, underscoring unmet medical needs. BioMarin is advancing clinical trials for hypochondroplasia, idiopathic short stature, and other growth conditions.
BioMarin presented four-year data from its Phase 3 study on ROCTAVIAN® at the ISTH 2024 Congress, showing long-term safety and efficacy in treating severe hemophilia A. The study revealed that 73.6% of patients had zero treated bleeds in year four, with stable FVIII levels. Mean FVIII activity was 27.1 and 16.1 IU/dL by OSA and CSA assays, respectively. Additionally, there was a notable improvement in health-related quality of life (HRQoL), with a 6.2 point increase in Haemo-QOL-A Total Score. Phase 2 results also showed seven-year bleed control for a majority of patients.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will be presenting at the BofA Securities 2024 Healthcare Conference on May 15, 2024, in Las Vegas, NV. Alexander Hardy, President, and CEO of BioMarin, will be presenting at 10:00am PT / 1:00pm ET. The presentation will be available via live audio webcast and archived on the company's website.
BioMarin Pharmaceutical Inc. presented positive new data at the 2024 Pediatric Endocrine Society Annual Meeting, highlighting the safety and efficacy of VOXZOGO® (vosoritide) in children with achondroplasia and other growth-related conditions. Results from multiple studies showed significant improvements in annualized growth velocity and height standard deviation, particularly in children with Noonan syndrome, ACAN deficiency, and NPR2 mutations. Data also demonstrated VOXZOGO's potential to positively impact growth beyond achondroplasia. Clinical trials for growth-related conditions are ongoing, with promising results for children with achondroplasia who initiated treatment during adolescence.
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