• BioMarin discontinued dosing and enrollment in its Phase 2 VOXZOGO trials for Turner syndrome, SHOX-deficiency and ACAN-deficiency after several slipped capital femoral epiphysis events occurred in two investigator-sponsored trials.
• SCFE events have not been observed in BioMarin’s Phase 2 trials in these conditions and have not been reported among more than 5,000 infants and children who received VOXZOGO for achondroplasia.
• BioMarin cited 10 years of clinical research and more than 10,000 patient-years of VOXZOGO safety data from clinical studies and post-marketing surveillance.
• The Phase 2 CANOPY trials of VOXZOGO in Noonan syndrome and idiopathic short stature without ACAN-deficiency will continue, with the latter group representing about 95% of children enrolled in the ISS trial.
• BioMarin said it uses its investor relations website, investors.biomarin.com, to disclose material non-public information and comply with Regulation FD.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. BioMarin Pharmaceutical Inc. published the original content used to generate this news brief via EDGAR, the Electronic Data Gathering, Analysis, and Retrieval system operated by the U.S. Securities and Exchange Commission (Ref. ID: 0001193125-26-107314), on March 16, 2026, and is solely responsible for the information contained therein.