On March 16, 2026, BioMarin Pharmaceutical Inc. announced its decision to discontinue dosing and enrollment in its Phase 2 trials for VOXZOGO in Turner Syndrome, SHOX-deficiency, and Aggrecan (ACAN)-deficiency. This decision follows the occurrence of several slipped capital femoral epiphysis (SCFE) events in two ongoing investigator-sponsored trials. Notably, SCFE events have not been observed in the Phase 2 BioMarin trials for these same conditions, nor have any cases been reported in the more than 5,000 infants and children who have received VOXZOGO for achondroplasia, which includes 10 years of clinical research and over 10,000 patient-years of safety data in clinical studies and post-marketing surveillance. Furthermore, there have been no observed cases in BioMarin’s clinical trials for hypochondroplasia. Despite this setback, the Phase 2 CANOPY trials of VOXZOGO in children with Noonan syndrome, as well as those living with idiopathic short stature (ISS) without ACAN-deficiency, will continue as planned. BioMarin uses its investor relations website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. The company emphasizes that these forward-looking statements involve risks and uncertainties, and actual results may differ materially from these statements. Investors are advised not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation to update or alter any forward-looking statement, whether as a result of new information, future events, or otherwise.

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