The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to dexpramipexole, its oral drug candidate for the treatment of hypereosinophilic syndrome (HES). HES is a rare and often incapacitating disorder with limited treatment options.
The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose, or prevent diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period against competition, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.
We are very pleased to receive orphan drug designation for dexpramipexole for HES, said Michael Bozik, M.D., CEO of Knopp Biosciences. There is an urgent need for effective treatments for patients suffering from this under- recognized, debilitating disease, especially for oral medications that selectively deplete eosinophils, and this designation represents an important regulatory milestone as we advance our dexpramipexole program toward late-stage clinical development.
Knopp is developing dexpramipexole as a targeted, oral therapeutic for eosinophilic inflammatory disorders. Eosinophils are white-blood cells that...