December 27, 2022
1 minute read
The FDA has issued a full response to Ipsen for its New Drug Application for palovarotene, an investigational treatment for reducing new abnormal bone formation in patients with fibrodysplasia ossificans progressis.
The letter from the FDA relates to the agency’s request for additional information about palovarotene trial data from October, Ipsen said in a press release. The company plans to respond to this request within the first quarter of 2023.
Fibrodysplasia ossificans progressive (FOP) is a rare disease that causes persistent and continuous bone formation in soft and connective tissues such as muscles, tendons and ligaments, says The Release. As bone accumulates in joints and other areas over time, FOP severely limits mobility and function.
“While this will extend the review timeline for palovarotene, we are continuing to work with the FDA to provide the requested information and to demonstrate that investigational palovarotene is a groundbreaking study that reduces new abnormal bone formation and slows the progression of FOP.” I believe it has the potential to be a cure.” Howard Mayer, Ipsen Executive Vice President and Head of Research and Development said:
According to the release, FOP affects an estimated less than 400 people in the United States and 900 worldwide. Due to bone formation abnormalities, FOP patients can permanently lose the ability to move joints in the neck, back, shoulders, chest, legs, and arms.
“Patients with FOP in the United States currently do not have an approved treatment option to slow disease progression, which remains a strong reason for us to pursue bringing this potential treatment option to FOP.” said Mayer.