Research & Development
Ipsen Pharma receives negative CHMP opinion for palovarotene as potential FOP treatment
26 May 2023 -

Biopharmaceutical company Ipsen (Euronext Paris:IPN) (ADR:IPSEY) revealed on Friday that it has received a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) regarding palovarotene as a potential treatment for fibrodysplasia ossificans progressiva (FOP).

This decision confirms the negative opinion given in January 2023.

Palovarotene is the first medicine submitted for regulatory approval for FOP. The disease causes continuous and permanent abnormal bone formation, leading to mobility loss and shortened life expectancy. No disease-modifying treatments are currently available in the EU.

Ipsen said that it remains committed to introducing a new treatment option for FOP and will continue regulatory processes in other countries, including the United States.

"We continue to believe that our Phase III MOVE trial, the first and largest study to be conducted in patients with FOP, has shown that palovarotene has the potential to reduce the new extra-skeletal bone formation caused by the disease and to slow its progression," said Howard Mayer, executive vice president and head of Research and Development for Ipsen.

Palovarotene is an investigational oral medicine targeting retinoic-acid receptor gamma. It received Orphan Drug and Breakthrough Therapy Designations from the US Food and Drug Administration (FDA) for the potential treatment of FOP, as well as orphan medicine designation from the EMA.