FDA Grants Ipsen Priority Review for Palovarotene in Patients with FOP

The U.S. Food and Drug Administration has accepted for Priority Review Ipsen’s resubmitted New Drug Application for its experimental therapy palovarotene for the treatment of patients with fibrodysplasia ossificans progressiva, an ultra-rare genetic disorder.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by bone that forms outside the normal skeleton, in muscles, tendons, or soft tissue. FOP has an estimated prevalence of 1.36 per million individuals globally; however, the number of confirmed cases varies by country. The median age at time of FOP diagnosis is five years old.

Palovarotene is an oral investigational, selective retinoic-acid receptor gamma (RARγ) agonist being developed as a potential treatment for people living with the debilitating ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP). Palovarotene, which received Rare Pediatric Disease and Breakthrough Therapy designations from the FDA for the potential treatment of FOP, was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019.

The palovarotene New Drug Application (NDA) was initially accepted by the FDA for Priority Review on May 28, 2021, and Ipsen announced withdrawal of the NDA on August 13, 2021. The resubmission of the palovarotene NDA has been accepted by the FDA for Priority Review in the U.S.

In January 2022, Health Canada was the first regulatory authority to approve Sohonos (palovarotene capsules) to reduce the formation of HO in adults and children aged 8 years and above for females and 10 years and above for males with FOP. Sohonos is approved in Canada for the treatment of patients with FOP for both chronic use and for flare-ups in these patient populations. Palovarotene is not currently approved outside of Canada. A Marketing Authorization Application for palovarotene has also been filed in the EU in 2021, with responses to questions raised by the European Medicines Agency submitted in June 2022, in line with the ongoing regulatory process. Ipsen anticipates additional applications to other regulatory agencies in due course.

The FDA’s Priority Review designation is assigned to investigational therapies that, if approved, represent a significant improvement in the safety or effectiveness of the treatment compared to the current standard of care.

“This FDA submission acceptance marks a significant milestone for those living with FOP in the U.S. where no approved treatment options exist today for this progressive and debilitating ultra-rare genetic disorder,” said Howard Mayer, executive vice president and head of research and development for Ipsen. “We remain committed to the FOP community, and we hope the Priority Review is one step closer towards getting a much-needed treatment to people living with FOP as soon as possible.”

Photo: Howard Mayer, executive vice president and head of research and development for Ipsen

Author: Rare Daily Staff