Rare Disease Report

Two Drugs Get Orphan Designation for Fibrodysplasia Ossificans Progressiva

AUGUST 20, 2015
Matthew Mahady
The US Food and Drug Administration (FDA) has granted La Jolla Pharmaceutical Company orphan drug designation for two novel compounds for fibrodysplasia ossificans progressiva.

The compounds are small-molecule kinase inhibitors designed to selectively block a specific member of the bone morphogenetic protein type-I receptor family, ALK2.  La Jolla recently entered an exclusive licensing agreement with Vanderbilt University to develop these compounds.

FOP is a rare genetic disorder in which the body abnormally develops bones in muscles. FOP is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway. In early childhood, people with the disorder develop soft tissue swellings that transform into bone. Development of these lesions is worsened by trauma and surgical intervention leads to dramatic and explosive new bone growth. The average survival rate of FOP patients is approximately 40 years. Death often occurs from complications of thoracic insufficiency syndrome.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a 7-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care compared with another drug of its type previously granted the designation for the same indication.

George Tidmarsh, MD, PhD, CEO and president of La Jolla said, “There are no known treatment options available for patients suffering from FOP and the orphan drug designations recognize the significant unmet need that exists within this disease.”

Other Treatment Options In Development for FOP

Last December, the FDA granted Fast Track designation to palovarotene, a retinoic acid receptor gamma agonist.

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 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.

In preclinical studies, treatment with palovarotene after injury prevented heterotopic bone formation in a mouse model of FOP. Because the safety of palovarotene in humans had already been established in clinical trials of patients with emphysema and because no treatments are available for FOP, the FDA allowed Clementia Pharmaceuticals to proceed straight to phase 2 clinical trials in patients with FOP.


La Jolla Pharmaceutical Company Receives Orphan Drug Designation for Two Novel Compounds for Fibrodysplasia Ossificans Progressiva [news release]. San Diego, CA: La Jolla Pharmaceuticals; August 18, 2015. http://www.businesswire.com/news/home/20150818005500/en/La-Jolla-Pharmaceutical-Company-Receives-Orphan-Drug#.VdWc6c7ney0

image courtesy wikimedia commons https://commons.wikimedia.org/wiki/File:Fibrodysplasia_ossificans_progressiva.jpg

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