Knopp Biosciences Receives FDA Orphan Drug Designation for Dexpramipexole for Treatment of Hypereosinophilic Syndrome

PITTSBURGH, PA, USA – April 24, 2019 – Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases with high unmet needs, announced today that 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 play a central role in severe diseases such as asthma and HES, and dexpramipexole has been shown to selectively, profoundly, and persistently reduce blood and tissue eosinophil levels in multiple clinical trials. Results of a Phase 2 study of dexpramipexole in HES have been presented to the American Society of Hematology and published in the journal Blood.


Knopp Biosciences, based in Pittsburgh, PA, USA, is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases with a high unmet need. Knopp’s clinical-stage small molecule, dexpramipexole, is entering Phase 2 clinical trials in eosinophilic asthma and Phase 3 development in hypereosinophilic syndrome. Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain.

Forward Looking Statement

This press release contains “forward-looking statements,” including statements relating to planned regulatory filings and clinical development programs. All forward-looking statements are based on management’s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp’s actual results to differ from our expectations. There can be no assurance that any investigational drug product will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market a product. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.

Knopp’s pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.

Media Contact: 
James Heins
Westwicke PR
Tel: (203)682-8251
Email: [email protected]