MONTREAL, Nov. 21, 2014 /PRNewswire/ -- 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.
For more information and answers to frequently asked questions about Clementia's clinical trials for palovarotene in FOP, please visit www.clementiapharma.com.
About Fibrodysplasia Ossificans Progressiva (FOP)
FOP is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in new, abnormal bone formation in muscles, tendons, and ligaments. Flare-ups begin early in life and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza infections. Recurrent flare-ups progressively restrict movement by locking joints, leading to cumulative loss of function and disability. FOP is caused by a point mutation in the ALK2/BMP Type I receptor; the mutation results in over-activity of the receptor. Virtually all known patients have the same point mutation and have congenital malformations of the big toes at birth. FOP is thought to affect less than one individual for every million lives.
Palovarotene is a retinoic acid receptor gamma agonist in-licensed from Roche Pharmaceuticals, where it was previously evaluated in more than 800 individuals including healthy volunteers and patients with chronic obstructive pulmonary disease. Palovarotene has been shown to block bone formation in a variety of mouse models of FOP and is being investigated as a potential treatment for FOP.
About Clementia Pharmaceuticals Inc.
Clementia is a privately held, clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people living with rare diseases. The company is advancing a novel retinoic acid receptor gamma agonist to address diseases of heterotopic ossification, including fibrodysplasia ossificans progressiva. For more information, please visit www.clementiapharma.com.
SOURCE Clementia Pharmaceuticals, Inc.