BioMimetic Therapeutics Receives Orphan Drug Designation for PDGF Treatment of Osteochondritis Dissecans
BY LAUREN UZDIENSKI, AUGUST 12, 2010
BioMimetic Therapeutics announced this morning that it has received orphan drug designation from the FDA for their PDGF technology in the treatment of osteochondritis dissecans (OCD) of the knee, elbow or ankle. PDGF is the same molecule utilized in the company's Augment bone graft, for which the company is seeking PMA approval in foot and ankle fusions and anticipating a panel hearing later this year or in early 2011.
Similar to an HDE designation for devices, orphan drugs address conditions affecting less than 200,000 people in the United States. The orphan drug designation can have a number of benefits, including an accelerated path to FDA approval, tax credits and seven years of market exclusivity.
The orphan drug program eases certain regulatory requirements, requiring less rigorous data (enrolling studies would be a challenge in such small populations), offering R&D credits and waiving certain fees associated with filing. The program has clearly been successful in advancing treatments for rare diseases, with the FDA noting on their website that since the law was enacted in 1983, more than 200 drugs and biological products addressing these small patient populations have been brought to market. In the decade prior to 1983, fewer than ten such products come to market.
Incentives aside, the orphan designation does limit the market for a treatment. One example is Stryker's OP-1, which was developed under an HDE (a regulatory exemption for devices intended to treat conditions affecting fewer than 4,000 U.S. patients annually) and has failed to gain approval for broader indications. The company has been dogged with allegations regarding off-label marketing and has received a federal indictment over the claims. Right now, the future for OP-1 is uncertain. At the FDA panel hearing last year, the agency emphasized that the PMA application was being evaluated on its own merits, independent of the product's HDE record, and the outcome of that meeting seemed to suggest that Stryker would have to start over with a new study if they wanted to continue to pursue posterolateral lumbar fusion labeling. In addition to that indication, at the time of the FDA panel, Stryker was conducting pilot studies on OP-1 for use in knee arthritis, degenerative disc disease and TLIFs, though the company has not commented publicly on OP-1 in some time.
