Ipsen announced that the European Commission has followed guidance provided by the Committee for Medicinal Products for Human Use (CHMP) in May this year, and has not granted marketing authorization for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva (FOP). Palovarotene 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.
“We worked tirelessly to bring a greatly needed treatment option to patients living with FOP in the E.U.,” said Howard Mayer, Executive Vice President and Head of Research and Development 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 submissions.”
Palovarotene was studied in a comprehensive clinical program over 15 years. This included MOVE, the first and largest Phase III clinical trial for FOP, a condition that causes abnormal bone growth. The average age of diagnosis for FOP is five years old and the average life expectancy is 56 years old. FOP is a chronic and progressive condition, where flare-ups occur that can lead to the development of new, abnormal bone formulation, accumulating outside of the skeleton in muscles, joints, and other areas of the body. As a result, most people living with FOP eventually lose the ability to eat and drink on their own. By the age of 30 years old, many will need a wheelchair to get around and full-time care. Life expectancy is shortened, as untimely death can be caused by bone formation around the ribcage, leading to breathing problems and cardiorespiratory failure.
“It is devastating to hear that the wait for an innovative treatment for people living with FOP, and their families and caregivers will continue, as this negative decision from the European Commission means that this treatment will not be made available to patients.” said Dr. Genevieve Baujat, Clinical Geneticist Consultant at Necker-Enfants Malades Hospital, Paris, France. “Many of the clinicians that manage patients with this disease in Europe will have participated in the MOVE clinical trial and have seen the potential for palovarotene.”