Posted by Michael Wonder on 11 Feb 2019
Clementia granted rare paediatric disease designation by FDA for palovarotene for fibrodysplasia ossificans progressiva
11 February 2019 - Clementia Pharmaceuticals today announced that the U.S. FDA has granted rare paediatric disease designation to palovarotene for the treatment of fibrodysplasia ossificans progressiva.
Palovarotene, an investigational therapy for fibrodysplasia ossificans progressiva (FOP), has previously been granted orphan drug, fast track and breakthrough designations by the FDA for FOP.
In October 2018, based on a meeting with the FDA, Clementia announced that the Agency was supportive of the company’s plan to submit a new drug application for palovarotene for the prevention of heterotopic ossification associated with flare up symptoms in patients with FOP. The FDA based its assessment on the efficacy and safety data generated in the completed Phase 2 clinical program. The company’s submission preparations are underway, with a submission targeted for the second half of 2019. If approved, Clementia expects a first commercial launch for palovarotene in 2020.
