Benzinga | Aug 4, 2020 07:51AM EDT

Eloxx Pharmaceuticals Receives US Orphan Drug Designation For ELX-02 For The Treatment Of Cystic Fibrosis

WALTHAM, Mass., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ:ELOX), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for ELX-02 for the treatment of Cystic Fibrosis.



The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan-drug designation qualifies Eloxx for certain benefits, including seven years of market exclusivity upon regulatory approval if received, exemption of FDA application fees, tax credits on qualified U.S. clinical trials and eligibility for grant funding opportunities that can be used for clinical trial costs.

"We are pleased to have received orphan drug designation from the FDA for ELX-02 in the U.S. Combined with our previous EMA orphan medicinal product designation in Europe, and upon approvals in the U.S. and the E.U., we will be able to obtain significant periods of marketing exclusivity in these important markets and provide treatment options to patients with high unmet medical needs, particularly in patients with nonsense mutations for whom there are few, if any, treatments available," said Dr. Gregory Williams, Chief Executive Officer of Eloxx Pharmaceuticals. "As previously announced, we have resumed enrollment in our Phase 2 proof of concept clinical trials in Israel and Europe, with our trial in the U.S. continuing to be temporarily paused due to the COVID-19 pandemic. We look forward to reporting top line results as soon as feasible, and believe that this will be a major value inflection point for our Company."