Clementia gets Orphan Medicinal Product Designation from EMA for palovarotene

Nov 21 2014

Clementia Pharmaceuticals, Inc. announced today that the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation for palovarotene, the company's lead product candidate, for the treatment of fibrodysplasia ossificans progressiva (FOP). FOP is a rare, severely disabling genetic disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and new abnormal bone formation. This process, known as heterotopic ossification (HO), occurs in muscles, tendons and ligaments, causing significant morbidities and progressive disability.

"FOP is an extremely rare, serious genetic condition for which there are no approved treatments," said Dr. Genevieve Baujat of Paris' Laboratoire de Genetique Moleculaire, Institut de Recherche and Hopital Necker-Enfants Malades and a principal investigator for palovarotene's Phase 2 clinical trial. "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."

Palovarotene, an investigational retinoic acid receptor gamma agonist, is currently in a Phase 2 clinical trial in patients with FOP and was previously designated an orphan drug by the U.S. Food and Drug Administration. Clementia recently announced the launch of a 12-month, open-label extension study for patients with FOP who complete the 12-week, randomized, double-blind, placebo-controlled study of palovarotene.

"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," said Chris Bedford-Gay, Founder of FOP Friends, a UK-based FOP charity and patient organization.

The EMA's orphan medicinal product exclusivity is designed to provide regulatory and financial incentives for companies to develop and market treatments for life-threatening or debilitating conditions of not more than five in 10,000 prevalence in the European Union (EU). Orphan medicinal products are granted 10 years of market exclusivity in the EU after receiving marketing authorization.

SOURCE Clementia Pharmaceuticals, Inc.