FDA Accepts NDA for First Potential Treatment for Fibrodysplasia Ossificans Progressiva

Ipsen announced that its New Drug Application (NDA) for palovarotene, an oral, investigational, selective RARγ agonist for the prevention of heterotopic ossification (new bone formation) as a potential treatment option for people living with the progressive disabling and ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP), has been accepted by the U.S. Food and Drug Administration (FDA). The target regulatory action date assigned by the FDA under a Priority Review status is November 30th, 2021.

FOP is an ultra-rare genetic disorder with an estimated prevalence of 1.36 per million individuals; however, the number of confirmed cases varies by country. It is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as heterotopic ossification (HO), which can be preceded by painful soft-tissue swelling or “flare-ups”. HO, once formed, is irreversible and leads to loss of mobility and shortened life expectancy.

Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen, said “With no approved treatments for this progressive and debilitating disease, there remains a great unmet medical need for the FOP community. This year marks 15 years since the discovery of the mutation in the gene ALK2/ACVR1 which causes FOP, and the palovarotene submission is the first worldwide for a potential treatment in this disease. Our teams at Ipsen are now focused on working closely with regulatory authorities to bring this potential treatment option to people living with FOP around the world. We want to thank all the people living with FOP, their families, caregivers and healthcare teams who have participated in the palovarotene clinical program.”