Eloxx Pharmaceuticals, Inc. (“Eloxx”), (NASDAQ:ELOX), announced on 4/27/18 that it has received orphan-drug designation from the Food and Drug Administration (FDA) for ELX-02 for treatment of cystinosis from the FDA.
“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.”
ELX-02 is designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length protein to restore activity of the mutated protein. A nonsense mucation is a point mutation in a sequence of DNA that results in a premature stop codon.
The European Medicines Agency (the “EMA”) has designated ELX-02 as an orphan medicine for the treatment of mucopolysaccharidosis type I (“MPS I”), and the FDA has also granted orphan drug designation to ELX-02 for the treatment of MPS I and for the treatment of Rett Syndrome.