FDA approves palovarotene capsules for fibrodysplasia ossificans progressiva

The U.S. Food and Drug Administration (FDA) has granted approval for palovarotene (Sohonos; Ipsen) capsules in adults and pediatric patients aged 8 years and older for females, and 10 years and older for males, who have fibrodysplasia ossificans progressiva (FOP).

The FDA approval was granted based on data from the Phase 3 MOVE trial, which is the largest multicenter, open-label trial involving both adult and pediatric patients. The study spanned 18 months and included 107 patients, representing approximately 12% of the estimated global FOP population.

Participants received oral palovarotene, and their outcomes were compared with untreated individuals from Ipsen’s worldwide FOP Natural History Study. The study results showed that palovarotene effectively reduced the annualized volume of heterotopic ossification when compared to no treatment beyond the standard of care. This reduction amounted to 54% according to a weighted linear mixed effect model.

The study demonstrated that palovarotene exhibited a well-characterized safety profile, with adverse events consistent with the broader systemic retinoid class. Common treatment-related adverse reactions reported in the study included mucocutaneous events such as dry skin, lip dryness, alopecia, drug eruption, rash, and pruritus, as well as musculoskeletal events such as arthralgia and premature closure of growth plates in growing children.

Read the full press release here.