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.
FUSE: Revolutionizing the analysis of gene mutations