Health
BioMarin Presents New Data Demonstrating Favorable Safety and Strong Adherence in Real-World Clinical Practice with VOXZOGO® (vosoritide) in Children Under 3 with Achondroplasia at 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ® (pegvaliase-pqpz) in adults with phenylketonuria (PKU). The latest findings further validate the long-term benefit and established safety profiles of both medicines, which continue to play a pivotal role for people living with these genetically defined condit
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"New data also to be presented for PALYNZIQ® (pegvaliaise-pqpz), reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with phenylketonuria","length":202,"tagName":"p","attribs":{}},{"type":"text","content":"SAN RAFAEL, Calif., March 20, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ® (pegvaliase-pqpz) in adults with phenylketonuria (PKU). The latest findings further validate the long-term benefit and established safety profiles of both medicines, which continue to play a pivotal role for people living with these genetically defined conditions.","length":566,"tagName":"p"},{"type":"image","alt":"BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)","displaySize":"","headline":null,"caption":"BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)","className":"","disableSlideshowImg":false,"size":{"original":{"width":400,"height":59,"url":"https://media.zenfs.com/en/prnewswire.com/db94610f2d68c7fd655942d2889db5d2"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/KxRqzB62ez2RdMQaPUuMVg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTcwNTtoPTEwNDtjZj13ZWJw/https://media.zenfs.com/en/prnewswire.com/db94610f2d68c7fd655942d2889db5d2","width":400,"height":59}},"href":"https://mma.prnewswire.com/media/534018/BioMarin_Pharmaceutical_Inc_Logo.html","hrefExternal":true,"rel":"nofollow"},{"type":"text","content":"The data will be presented at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Los Angeles.","length":144,"tagName":"p"},{"type":"text","content":"Data Highlight VOXZOGO Treatment Adherence and Favorable Safety Profile in Infants","length":82,"tagName":"p"},{"type":"text","content":"New data from a study in Japan showed strong treatment adherence with VOXZOGO in children with achondroplasia under the age of 3, with no reported treatment-related adverse events nor any dose interruptions among 63 children followed for up to 23.7 months. These real-world findings validate VOXZOGO's established safety profile and reinforce the therapeutic benefit seen in clinical studies. The stud...