EMA Grants Orphan Status To Clementia Pharma's Lead Drug

Privately-held, clinical stage biopharmaceutical firm Clementia Pharmaceuticals announced that it has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for its lead drug palovarotene as treatment for fibrodysplasia ossificans progressiva (FOP).

Palovarotene is a retinoic acid receptor gamma agonist that the company has in-licensed from Roche, where it has been assessed in over 800 patients with chronic obstructive pulmonary disease (COPD). Clementia has conducted a midstage trial for palovarotene in FOP earlier this year. The company also announced an extension study of the Phase 2 trial for the drug last month to investigate long-term safety and efficacy.

Fibrodysplasia ossificans progressive is a genetic disease that causes painful soft tissue swelling and abnormal bone formation known as heterotopic ossification (HO). The disease affects the muscles, ligaments, and tendons. FOP results from a point mutation caused by overactivity in the ALK2/BMP Type I receptor. The disease is considered an orphan condition, as it affects less than 1 in 1 million.

“FOP is an extremely rare, serious genetic condition for which there are no approved treatments. The EMA's orphan medicinal product designation encourages companies like Clementia to make the significant investment of time, effort, and money to develop a treatment,” said principle investigator Dr. Genevieve Baujat of Paris' Laboratoire de Genetique Moleculaire.

Chris Bedford-Gay, Founder of charity and patient organization FOP Friends, was pleased with the designation. “People with FOP, including my eldest son, and their loved ones currently live with no viable treatment options, and it is extremely heartening to see progress in bringing a potential therapy to our community.”

The EMA’s orphan designation is granted to drugs that address life-threatening or disabling conditions affecting no more than 5 per 10,000 in the EU. Upon approval, orphan drugs are awarded 10 years of market exclusivity. Palovarotene previously received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in July.