EC Rejects Ipsen FOP Drug Palovarotene

Rare Daily Staff

The European Commission said it would not grant marketing authorization approval to Ipsen’s therapy for the ultra-rare bone formation condition fibrodysplasia ossificans progressive.

The decision followed a negative opinion by the Committee for Medicinal Products for Human Use in May. Palovarotene is the first treatment to be submitted anywhere in the world for regulatory approval for fibrodysplasia ossificans progressive (FOP).

FOP is a chronic and progressive condition, where flare-ups occur that can lead to the development of new, abnormal bone formation, 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.

There are no currently approved options available in the European Union for the treatment of FOP, an ultra-rare condition, that continuously and permanently causes abnormal bone formation, leading to progressive mobility loss and shortened life expectancy.

Palovarotene is an experimental, oral medicine that selectively targets the retinoic-acid receptor gamma (RARγ), which is an important regulator of skeletal development and ectopic bone in the retinoid signaling pathway. Palovarotene is designed to mediate the interactions between the receptors, growth factors, and proteins within the retinoid signaling pathway to reduce new abnormal bone formation.

Palovarotene received Orphan Drug and Breakthrough Therapy designations from the U.S. Food and Drug Administration for the potential treatment of FOP and was granted Priority Review. It remains under FDA review, with agency action on its application expected by August 16.

Palovarotene was also granted orphan medicine designation by the European Medicines Agency. Palovarotene is currently authorized for use in appropriate patients only in Canada and provisionally in the U.A.E. where it is marketed as Sohonos.

Palovarotene was studied in a comprehensive clinical program over 15 years. This included MOVE, the first and largest phase 3 clinical trial for FOP. The average age of diagnosis for FOP is five years old and the average life expectancy is 56 years old.

“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.”