Ipsen’s Sohonos (palovarotene) has become the first drug approved for patients with fibrodysplasia ossificans progressiva (FOP) in the US, following the granting of a marketing authorisation by the Food and Drug Administration (FDA).
The capsules are approved for reduction in the volume of new heterotopic ossification (extra-skeletal bone formation) in adults and children aged eight years and older for females, and 10 years and older for males.
FOP is a rare, autosomal dominant disease where connective tissue such as muscle, tendons and ligaments gradually turn into bone tissue, causing limited movement, deformities and severe disability. It affects an estimated 400 people in the US and 900 people globally.
The FDA approval is based on data from the Phase III MOVE trial, which demonstrated that palovarotene effectively reduced annualised heterotopic ossification (HO) volume compared with no treatment beyond standard of care (54% reduction with weighted linear mixed effect model).
“As a clinician caring for patients with FOP, I personally see the daily challenges and stresses that our patients and their families must contend with,” said Dr Edward Hsiao, Professor of Medicine, Division of Endocrinology and Metabolism, University of California, San Francisco. “The published Phase III MOVE study showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with FOP.
“Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. However, since the accumulation of HO in FOP is progressive, irreversible, and life altering, this medication is an important treatment option for our FOP community.”
Sohonos received a priority review, fast track designation and breakthrough therapy designation from the FDA for this indication. The EU’s regulator chose not to recommend the drug in January 2023.
Edited by Diana Spencer, Senior Digital Content Editor, Drug Discovery World