BMRN Biomarin Pharmaceutical Inc - 8-K

Item 8.01 Other Events.

On March 16, 2026, BioMarin Pharmaceutical Inc. (“BioMarin”) announced its decision to discontinue dosing and enrollment in its Phase 2 trials for VOXZOGO in Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency following the occurrence of several slipped capital femoral epiphysis (SCFE) events in two ongoing investigator-sponsored trials. SCFE events have not been observed in the Phase 2 BioMarin trials in these same conditions, nor have any cases been observed in the more than 5,000 infants and children who have received VOXZOGO for achondroplasia, which includes 10 years of clinical research and more than 10,000 patient-years of safety data in clinical studies and post-marketing surveillance. There have similarly been no observed cases in BioMarin’s clinical trials for hypochondroplasia. 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, which represents approximately 95% of children enrolled in the ISS trial, will continue as planned.

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Forward-Looking Statements

This Current Report on Form 8-K contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin’s decision to discontinue enrollment and dosing of VOXZOGO in children with Turner syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency in BioMarin’s Phase 2 CANOPY clinical trials of VOXZOGO; and plans to continue 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. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, the results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; the content and timing of decisions by regulatory authorities; and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption “Risk Factors” in BioMarin’s Annual Report on Form 10-K for the year ended December 31, 2025, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.