EC rejects Ipsen's palovarotene for the treatment of FOP

Not surprisingly given the negative opinion from the European Medicines Agency’s (EMA) human medicines committee (CHMP), the European Commission has followed guidance and has not granted marketing authorization for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva (FOP).

Under development at French mid-sized pharma company Ipsen (Euronext: IPN) palovarotene, a novel RARγ agonist, is the first treatment to be submitted anywhere in the world for regulatory approval for FOP, an ultra-rare disease with approximately 900 known cases worldwide, the firm noted.

“We worked tirelessly to bring a greatly needed treatment option to patients living with FOP in the EU,” said Howard Mayer, executive vice president and head of R&D for Ipsen. “We believe that our clinical data provide evidence supporting the effect of palovarotene on the reduction of new, abnormal bone formation, known as heterotopic ossification, which characterizes the disease. We are therefore disappointed that the European Commission decided not to approve this treatment for patients with FOP in Europe. We have learned through this experience, and we have been motivated by the support of the FOP community, including those living with the condition and the doctors and healthcare providers managing their care. This has encouraged us as we continue to progress with other regulatory submission,” he added.