Reports of hip joint complications prompt suspension of studies, sending shares lower
Shares of BioMarin Pharmaceutical (BMRN) fell roughly 4% after the company announced it would discontinue mid-stage clinical trials of its bone disorder treatment Voxzogo for several rare genetic conditions following reports of safety concerns among patients.
The company had been testing the therapy in individuals with Turner Syndrome, SHOX Deficiency, and Aggrecan Deficiency—three genetic disorders associated with severe short stature and, in some cases, abnormal bone development. Turner syndrome occurs in females missing part or all of one X chromosome, while SHOX deficiency is linked to a faulty gene responsible for growth regulation. Aggrecan deficiency stems from mutations affecting cartilage structure and skeletal development.
During the mid-stage studies, several patients developed a condition known as Slipped Capital Femoral Epiphysis (SCFE), a rare but serious complication in which the ball of the hip joint slips out of place from the thigh bone at the growth plate. The condition typically occurs in adolescents while bones are still developing and can lead to pain, mobility problems, and the need for surgical intervention.
Following these reports, BioMarin decided to halt the trials for those specific indications as a precautionary measure. Despite the setback, the company stated that it will continue evaluating Voxzogo in other patient populations, including individuals with Noonan Syndrome and patients experiencing unexplained forms of short stature.
Voxzogo already has an established regulatory foothold. The U.S. Food and Drug Administration approved the injectable therapy in 2021 for patients with Achondroplasia, the most common form of dwarfism.
Competition in the space has also intensified. Last month, Ascendis Pharma received FDA approval for Yuviwel, a once-weekly injection that became the second therapy authorized in the United States to treat achondroplasia.
While BioMarin’s decision highlights the challenges of developing therapies for rare genetic growth disorders, the company remains focused on advancing Voxzogo in other clinical areas where it believes the treatment may still provide meaningful benefits for patients.
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