BioMarin stops mid-stage trials of bone disorder treatment

BioMarin Pharmaceutical

16/03/2026 13:34

March 16 (Reuters) - BioMarin Pharmaceutical BMRN.O will discontinue mid-stage trials of its bone disorder treatment after reports of several cases of patients' hip joint slipping out of place, the drugmaker said on Monday, sending its shares down 4%.

BioMarin was testing its drug Voxzogo in patients with Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency, which are distinct genetic conditions that causes severe short stature and in some cases abnormal bone development.

Turner syndrome occurs in females missing part or all of an X chromosome, SHOX deficiency results from a missing or faulty growth-related gene, and ACAN deficiency is caused by mutations in a gene important for cartilage structure.

During the mid-stage trials, several patients faced slipped capital femoral epiphysis, where the ball of the hip joint slips off the thigh bone at the area where the bone is still growing.

BioMarin said it will continue testing the drug in patients with Noonan syndrome where typical development stops in various parts of the body, and in patients with a form of unexplained short stature.

The U.S. Food and Drug Administration approved the injection Voxzogo in 2021 for patients with achondroplasia, a form of dwarfism.

Last month, the FDA approved Ascendis Pharma's A71.F once-weekly injection Yuviwel, making it the second treatment for achondroplasia to gain U.S. approval.

 (Reporting by Christy Santhosh in Bengaluru; Editing by Sahal Muhammed)

 ((Christy.Santhosh@thomsonreuters.com;))