Positive outcome of FDA Advisory Committee on investigational palovarotene for fibrodysplasia ossificans progressiva- Ipsen.

The FDA is currently reviewing the New Drug Application (NDA) for palovarotene with a decision anticipated by August 16, 2023. If approved, palovarotene will be the first treatment in the U.S. for FOP.

The advisory committee voted 10 for and 4 against that evidence from the Phase III MOVE study show palovarotene is an effective treatment in patients with the ultra-rare bone disease fibrodysplasia ossificans progressiva (FOP). The committee further voted 11 for and 3 against on the benefits of palovarotene outweighing the risks, for the treatment of patients with FOP. FOP is an ultra-rare disease that causes permanent and continuous bone growth in soft and connective tissues like muscles, tendons and ligaments, also known as heterotopic ossification or HO. FOP impacts the lives of an estimated 400 people in the U.S. and 900 people globally.

As the disease continuously progresses with flare-up episodes causing rapid bone growth, FOP severely restricts mobility and function. Most people living with FOP inevitably lose the ability to eat and drink on their own, can not provide self-care or use the restroom themselves, and are unable to maintain employment. By the age of 30, most people with FOP require a wheelchair and full-time caregiver assistance. Without disease-modifying treatments, current management is limited to palliative care.