Eloxx Pharmaceuticals Announces FDA Grant of Orphan-Drug Designation for Investigational Drug ELX-02 for Treatment of Cystinosis

Eloxx Pharmaceuticals (NASDAQ:ELOX), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that it has received orphan-drug designation from the Food and Drug Administration (FDA) for ELX-02 for treatment of cystinosis from the FDA and that it will ring the Nasdaq Closing Bell on Monday, April 30, 2018.

As quoted in the press release:

“We are pleased to have received an orphan-drug designation for ELX-02 for treatment of cystinosis and for the opportunity to ring the NASDAQ Closing Bell in celebration of our recent listing on the Nasdaq coincident with our successful $50 million public offering,” said Robert E. Ward, Chairman and CEO of Eloxx Pharmaceuticals. “As we ring the closing bell, I would like to acknowledge and thank the employees, investors, patients, and partners who have supported Eloxx in our journey thus far. Given the clinical progress in 2017 for our lead product candidate, ELX-02, we are poised to seek regulatory clearance to initiate Phase 2 clinical trials in cystic fibrosis and cystinosis this year in Belgium and the United States, respectively. Our listing on the Nasdaq and capital raise position us well to advance these and other future clinical programs and pursue our mission, which is to transform the lives of patients with rare and ultra rare diseases.”

Click here to read the full press release.