FDA approves Sohonos capsules, the first and only treatment for people with fibrodysplasia ossificans progressiva

“The FDA approval of Sohonos is a breakthrough for the U.S. FOP community. For the first time doctors have an approved medicine available to them, shown to reduce the formation of new, abnormal bone growth, known as heterotopic ossification (HO), which causes debilitating mobility challenges and has a devastating impact on the lives of people with FOP,” said Howard Mayer, Head of Research and Development, Ipsen. “Development of medicines for rare diseases takes commitment and belief from everyone involved. We at Ipsen are sincerely grateful to the FOP community of patients and medical experts, as the first-ever treatment in the U.S. for managing FOP would not be possible without their participation in the clinical trials and ongoing support.”

The FDA approval is based on the pivotal efficacy and safety data from the Phase III MOVE trial, the first and largest multicenter, open-label trial in adult and pediatric patients. The 18-month data published in the Journal of Bone and Mineral Research, included 107 patients (12 percent of the estimated number of individuals worldwide living with FOP) who received oral palovarotene compared with untreated individuals from Ipsen’s global FOP Natural History Study. The study results demonstrated palovarotene effectively reduced annualized heterotopic ossification volume compared with no treatment beyond standard of care, (54% reduction with weighted linear mixed effect model). The study also demonstrated that palovarotene has a well-characterized safety profile, with adverse events consistent with the systemic retinoid class. The most common treatment emergent adverse reactions reported in the study were mucocutaneous events such as dry skin, lip dryness, alopecia, drug eruption, rash, and pruritus and musculoskeletal events such as arthralgia and premature growth plate closure in growing children.

See-"Reduction of New Heterotopic Ossification (HO) in the Open-Label, Phase III MOVE Trial of Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP)."; Robert J. Pignolo, Edward C. Hsiao, Mona Al Mukaddam, Geneviève Baujat, Staffan K. Berglund,et al., First published: 30 December 2022 https://doi.org/10.1002/jbmr.4762.