Ipsen S.A. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorization for investigational palovarotene as a treatment for the ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP). In the E.U. there are currently only symptomatic treatments for FOP, which do not reduce the formation of extra-skeletal bone in patients with the condition. Ipsen will be requesting a re-examination of the CHMP opinion, based on scientific data available from the existing palovarotene clinical trial program.

FOP causes permanent and continuous new bone formation in soft and connective tissues, like muscles, tendons and ligaments, a process known as heterotopic ossification (HO). Once formed, it is irreversible. The average age of diagnosis is 5 years old and ultimately FOP shortens the median life expectancy to 56 years as untimely death is caused by bone formation around the ribcage leading to breathing problems and cardiorespiratory failure.

FOP has an estimated prevalence of 1.36 per million individuals and about 900 people are diagnosed worldwide; however, the number of confirmed cases varies by country. The CHMP opinion is based on its review of data from MOVE, the first and larger Phase III efficacy and safety trial conducted in FOP. The primary objectives of MOVE were to evaluate the efficacy of palovarotene in reducing new HO volume, as assessed using whole-body computed tomography, compared with patients untreated beyond standard of care from Ipsen's global FOP natural history study, and to evaluate the safety of investigational palovarotene in adult and pediatric patients with FOP.