$55.140
+0.772 (+1.40%)收盤時
BMRN 資訊
BMRN 事件
BioMarin Appoints Cara Bauer as Chief People Officer
BioMarin Pharmaceutical announced that Cara Bauer, Ph.D., has been named Executive Vice President, Chief People Officer. Bauer will assume responsibility for the company's Human Resources organization, effective Oct. 5, succeeding Amy Wireman, who is retiring. Bauer joins BioMarin from Kyverna Therapeutics where she served as Chief Human Resources Officer.
BioMarin Presents POMBILITI + OPFOLDA Data in Japan
BioMarin Pharmaceutical announced new data evaluating the efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease were presented at the 31st Annual Congress of the World Muscle Society, in Hiroshima, Japan. These results from the PROPEL open-label extension study of POMBILITI + OPFOLDA, which were shared in a late-breaking presentation, demonstrated durable motor function outcomes and relative stabilization of pulmonary function over long-term follow-up. The analysis included data from 82 individuals who received POMBILITI + OPFOLDA continuously from baseline observed in the initial PROPEL study to year five. For the enzyme replacement therapy-experienced cohort, the mean duration of prior ERT at baseline was 7.6 years. These data represent long-term benefits for people with LOPD followed in the study, including both ERT-experienced and ERT-naive cohorts. "These five-year results demonstrate that POMBILITI and OPFOLDA can provide durable, long-term benefits for people living with late-onset Pompe disease, including those who had been on enzyme replacement therapy for years before enrolling in the study," said Greg Friberg, Chief Research & Development Officer. "Sustaining motor function and limiting pulmonary decline for up to five years in a progressive disease like LOPD is clinically meaningful, and we remain committed to continuing to share long-term evidence to inform the care of people with this rare genetic condition." No new safety signals were identified. 51.2% of participants experienced treatment-related adverse events, the majority of which were manageable. Five individuals discontinued treatment for safety reasons, including two due to serious treatment-related adverse events. Additional presentations at WMS included data evaluating nivudirsen an investigational antisense oligonucleotide for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy amenable to exon 51 skipping. Included in these presentations was an update from the ongoing Phase 1/2 351-201 study, which was previously shared at the 19th International Congress on Neuromuscular Diseases in July. Results from 351-201 showed treatment with nivudirsen was generally well tolerated and led to consistently positive outcomes across multiple measures of therapeutic benefit, including expression of near full-length dystrophin.
BioMarin Appoints Robert Plenge to Board of Directors
BioMarin Pharmaceutical (BMRN) announced that the company has appointed Robert Plenge, M.D., Ph.D., to the company's Board of Directors, effective September 10. Dr. Plenge is Executive Vice President, Chief Research Officer and Head of Research at Bristol Myers Squibb (BMY).
BioMarin Releases Phase 3 Study Data for VOXZOGO
BioMarin Pharmaceutical announced new data from the Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO, or vosoritide, in children with hypochondroplasia were published in New England Journal of Medicine Evidence and presented at the European Society for Paediatric Endocrinology 2026 Annual Meeting. The data included new results on the magnitude of benefit seen in children receiving VOXZOGO, including statistically significant improvements in annualized growth velocity, or AGV, standing height, height Z-score and arm span after 52 weeks, with safety findings consistent with the established profile of VOXZOGO in achondroplasia. The CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint. Children treated with VOXZOGO also showed significant improvements in standing height, height Z-score, and arm span compared with placebo. Children who received VOXZOGO also demonstrated numerical improvements in quality of life, and follow-up will continue to assess the impact of treatment over a longer term. The overall safety profile was consistent with previous studies of VOXZOGO, with most adverse events reported as mild and no treatment-related serious adverse events identified. BioMarin recently submitted its supplemental New Drug Application to the FDA for the approval of VOXZOGO for the treatment of hypochondroplasia and are on track with the submissions to the European Medicines Agency and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.
本頁僅供研究參考，不構成投資建議。模型可能出錯。過往表現不代表未來結果。