FDA Accepts for Priority Review Ipsen’s NDA for Palovarotene

Palovarotene is being reviewed as a therapy for an ultra-rare disease that transforms the body’s soft tissue into bone. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of Dec. 29, 2022.

The FDA has accepted for priority review Ipsen’s resubmitted new drug application (NDA) for investigational palovarotene for the treatment of patients with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disorder. FOP is disorder in which bone develops in areas where it is not normally present, which is called heterotopic ossification. It can cause skeletal muscles and soft tissue to transform into bone.

Ipsen is seeking approval of palovarotene, an oral, selective retinoic-acid receptor gamma (RARγ) agonist for the prevention of heterotopic ossification.

The FDA has assigned Dec. 29, 2022, as the Prescription Drug User Fee Act (PDUFA) goal date, which is on track with anticipated regulatory submission timelines.

“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,” Howard Mayer, M.D., executive vice president and head of research and development, Ipsen, said in a press release.

The palovarotene NDA was initially accepted by the FDA for priority review on May 28, 2021, and Ipsen announced withdrawal of the NDA on Aug. 13, 2021, after a discussion with regulators. Additional analyses and evaluation of data collected from Ipsen’s phase III MOVE and FOP program would be required to complete the review process. It was agreed between Ipsen and the FDA that it would not be possible to complete this within the current NDA review cycle.

In January 2022, Health Canada was the first regulatory authority to approve Sohonos (palovarotene capsules) to reduce the formation of heterotopic ossification in adults and children aged 8 years and above for females and 10 years and above for males with FOP. A Marketing Authorization Application for palovarotene has also been filed in the European Union. in 2021, with responses to questions raised by the European Medicines Agency submitted in June 2022.