BioMarin Pharmaceutical Inc.·Healthcare

BMRN's shares rise after Voxzogo hits the main goal in a late-stage hypochondroplasia study, supporting planned FDA filings in 2026.

BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) stock is trading higher on Thursday as the company is celebrating positive results from its Phase 3 CANOPY-HCH-3 study for VOXZOGO (vosoritide) in children with hypochondroplasia.

BioMarin Pharmaceutical said on Wednesday its treatment for a rare condition that leads to short stature helped boost growth in children, meeting the main goal of a late-stage study and sending shares up nearly 5% in extended trading.

Study met primary endpoint, exceeding expectations with a highly statistically significant improvement in annualized growth velocity (AGV, change from baseline) of 2.33 cm/yr compared to placebo at week 52, as well as statistically significant increases in standing height and height Z-score Study demonstrated statistically significant improvement in arm span at week 52 compared to placebo, a key measure linked to functional independence Safety findings consistent with the established profile in achondroplasia with no new safety signals identified Supplemental New Drug Application (sNDA) submission to U.S. Food and Drug Administration (FDA) planned for third quarter, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities Full data to be presented at an upcoming medical meeting SAN RAFAEL, Calif., May 20, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO ® (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualized growth velocity (AGV) compared to placebo (LS mean difference +2.33 cm/yr, p

BioMarin shares fall after BMN 401 meets a biomarker goal but misses a key radiographic endpoint in a late-stage ENPP1 deficiency study.

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BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; BMN 307, an AAV5 mediated gene therapy, which is in Phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; and BMN 255 that is in Phase 1/2 clinical trial for treating primary hyperoxaluria. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. The company was incorporated in 1996 and is headquartered in San Rafael, California.
Healthcare
Biotechnology
3,040
1999-07-26
0.23