BioMarin has halted dosing and enrollment in Phase 2 VOXZOGO trials for Turner syndrome, SHOX-deficiency, and ACAN-deficiency after SCFE events were reported in investigator-sponsored studies, while other CANOPY trials continue.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
BioMarin Pharmaceutical Inc. has discontinued dosing and enrollment in certain Phase 2 clinical trials evaluating VOXZOGO (vosoritide) after reports of slipped capital femoral epiphysis (SCFE) events in investigator-sponsored studies. The decision effects studies investigating the therapy in Turner syndrome, SHOX-deficiency, and Aggrecan (ACAN)-deficiency, according to a company filing with the U.S. Securities and Exchange Commission.
The company stated that several SCFE events were reported in two ongoing investigator-sponsored trials studying VOXZOGO in these conditions. As a precautionary measure, BioMarin has halted enrollment and dosing in its own Phase 2 trials for these indications while further evaluating the safety findings.
BioMarin noted that no SCFE cases have been observed in its own Phase 2 studies for these same indications. The company also emphasized that the event has not been reported among more than 5,000 infants and children treated with VOXZOGO for achondroplasia, representing approximately 10 years of clinical research and over 10,000 patient-years of safety data from clinical studies and post-marketing surveillance.
The therapy’s development in other growth-related conditions will continue. BioMarin confirmed that Phase 2 CANOPY trials evaluating VOXZOGO in children with Noonan syndrome and idiopathic short stature (ISS) without ACAN-deficiency will proceed as planned. The ISS group without ACAN-deficiency represents about 95% of children enrolled in that trial, according to the company.
VOXZOGO is currently approved in multiple regions for the treatment of achondroplasia, a genetic disorder that causes impaired bone growth. The drug works by targeting the C-type natriuretic peptide (CNP) pathway, helping regulate bone growth by counteracting abnormal FGFR3 signaling.63+
The development update was disclosed in a Form 8-K filing dated March 16, 2026, and the safety-related trial halt has also been confirmed in reporting by Fierce Pharma, which highlighted the company’s precautionary move following the SCFE events in external studies.
BioMarin said it will continue monitoring safety data as the evaluation progresses and maintain ongoing trials where no related safety signals have been observed.
Reference
Form 8-K, Biomarin Pharmaceuticals, United Staes Security and Exchange Commission, https://www.sec.gov/Archives/edgar/data/1048477/000119312526107314/d114815d8k.htm
BioMarin pumps brakes on 3 Voxzogo studies as competition in drug’s flagship achondroplasia indication mounts, 16 March 2026, https://www.fiercepharma.com/pharma/biomarin-pumps-brakes-3-voxzogo-studies-competition-drugs-flagship-achondroplasia-indication
About the Writer
Chikkula Pavan Kumar, PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.
