MONTREAL, Dec. 1, 2014 /CNW/ -- Clementia Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to palovarotene, which is being developed 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.
Established under the Food and Drug Administration Modernization Act of 1997 (FDAMA), Fast Track designation is designed to facilitate the development and expedite the review of drugs and biologic products that are intended to treat serious or life-threatening conditions, and that demonstrate the potential to address unmet medical needs. Fast Track designation emphasizes the critical nature of early and frequent communication between the FDA and the sponsoring company to improve the efficiency of product development.
"The receipt of Fast Track designation is an important milestone for palovarotene, as it reflects the FDA's recognition of the potential promise of this novel investigational therapy as a treatment for FOP," commented Clarissa Desjardins, Ph.D., Chief Executive Officer of Clementia. "We are actively engaging with the FDA as we proceed with our development of palovarotene, with the goal of expediting approval so we can meet the needs of this underserved patient population."
Palovarotene, an investigational retinoic acid receptor gamma agonist, is currently in Phase 2 of clinical development. Clementia recently announced the launch of a 12-month, open-label extension study for patients with FOP who complete a 12-week, randomized, double-blind, placebo-controlled study of palovarotene. 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 overactivity 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.
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SOURCE Clementia Pharmaceuticals, Inc.
For further information: Investor/Media Contact: Smitha Dwarakanath, SmithSolve LLC, 973-442-1555 ext. 122, http://clementiapharma.com