FDA accepts filing of palovarotene for fibrodysplasia ossificans progressiva.- Ipsen

The target regulatory action date assigned by the FDA under a Priority Review status is 30 November 2021. The NDA for palovarotene is primarily based on data from the ongoing MOVE trial, the first global multi-center Phase III trial in FOP. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of a chronic/flare-up dosing regimen of palovarotene in decreasing new annualized HO volume in patients with FOP. Post hoc analyses of the primary endpoint from the trial demonstrated a 62% reduction in mean annualized new HO volume in participants treated with palovarotene (8,821 mm3) (n=97) versus untreated participants from a natural-history study (23,318 mm3) (n=98) (nominal weighted linear mixed effects [wLME] model est. –11,611mm3, p-value = 0.0292). Overall, 29.3% of participants reported at least one serious adverse event (AE), including premature physeal closure (PPC) or epiphyseal disorder in 27.1% of the participants who were skeletally immature at baseline.4 As of the data cut-off, the most common treatment related AEs included skin and subcutaneous tissue disorders (97%), gastrointestinal disorders (77.8%), and infections and infestations (74.7%).