FDA issues complete response letter for rare genetic disease treatment

The FDA has issued a complete response letter to Ipsen for its new drug application for palovarotene, an investigational treatment for the reduction of new abnormal bone formation in people with fibrodysplasia ossificans progressiva.

The letter from the FDA is related to the agency’s request for additional information on palovarotene trial data from October, Ipsen stated in a press release. The company anticipates responding to the request within the first quarter of 2023.

Fibrodysplasia ossificans progressiva (FOP) is a rare disease that causes permanent and continuous bone formation in soft and connective tissue, such as muscles, tendons and ligaments, the release stated. With bone accumulating in joints and other areas over time, FOP severely restricts mobility and function.

“Although this extends the review timeline for palovarotene, we continue to work with the FDA to provide the requested information and believe that investigational palovarotene has the potential to be an innovative treatment to reduce new abnormal bone formation to slow the progression of FOP,” Howard Mayer, executive vice president and head of research and development at Ipsen, said in the release.

According to the release, FOP affects less than an estimated 400 people in the United States and 900 people globally. Due to abnormal bone formation, those with FOP may lose the permanent ability to move their neck, back, shoulders, chest, legs and arm joints.

“Currently, people living with FOP in the U.S. have no approved treatment option to slow the progression of the disease, and this remains our reason for being steadfast in our pursuit of bringing this potential treatment option for FOP,” Mayer said.