BioMarin stops mid-stage trials of bone disorder treatment

March 16 (Reuters) – BioMarin Pharmaceutical 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 withTurner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency,which are distinct genetic conditions that causes severe shortstature and in some cases abnormal bone development. * Turner syndrome occurs in females missing part or all ofan X chromosome, SHOX deficiency results from a missing orfaulty growth-related gene, and ACAN deficiency is caused bymutations in a gene important for cartilage structure. * During the mid-stage trials, several patients facedslipped capital femoral epiphysis, where the ball of the hipjoint slips off the thigh bone at the area where the bone isstill growing. * BioMarin said it will continue testing the drug inpatients with Noonan syndrome where typical development stops invarious parts of the body, and in patients with a form ofunexplained short stature. * The U.S. Food and Drug Administration approved theinjection Voxzogo in 2021 for patients with achondroplasia, aform of dwarfism. * Last month, the FDA approved Ascendis Pharma’s once-weeklyinjection Yuviwel, making it the second treatment forachondroplasia to gain U.S. approval.

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