Failed COPD Drug Being Tested for Fibrodysplasia Ossificans Progressiva

James Radke

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to palovarotene, a retinoic acid receptor gamma agonist, for the treatment of fibrodysplasia ossificans progressiva (FOP).
Palovarotene is in-licensed to Clementia Pharmaceuticals from Roche Pharmaceuticals.  At Roche, it had been investigated as a possible treatment for chronic obstructive pulmonary disease (COPD) but was not found to be effective in clinical trials. However, Clementia became interested in the drug when it was shown to block bone formation in a variety of mouse models of FOP. 
FOP is a 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. Recurrent flare-ups progressively restrict movement, leading to cumulative loss of function and disability. FOP is an ultra rare condition, affecting less than one in one million people.
Clarissa Desjardins, Ph.D., Chief Executive Officer of Clementia  said:

“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.”
“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.”  

A phase 2 clinical trial in patients with FOP is currently underway, including an open label extension of its 12 week, phase 2 study.
For more information about FOP, visit or

Image courtesy of wikimedia commons (cropped).
Printer Printing...