NESS ZIONA, Israel and WALTHAM, Massachusetts, November 7, 2016 /PRNewswire/ -- Eloxx Pharmaceuticals, a biotechnology company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations, announced today that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) has granted orphan drug designation to its lead propriety drug candidate, ELX-02 for the treatment of mucopolysaccharidosis type 1 (MPS I). Pedro Huertas, MD, PhD, Chief Medical Officer at Eloxx commented that "This Orphan Drug designation granted by the FDA and EMA recognizes the significant work achieved by Eloxx Pharmaceuticals in providing scientific and preclinical evidence about potential to become a transformational therapy for patients affected with MPS1 caused by nonsense mutations." . Silvia Noiman, PhD, Chief Executive Officer at Eloxx stated that "This is an important milestone for the company and a recognition for the hard work of the Eloxx team." ELX-02 is a synthetic designer aminoglycoside optimized as a translational read-though drug (TRID) for the treatment of genetic diseases caused by nonsense mutations. The compound is currently under investigation in Phase 1 first in human (FIH) clinical study in healthy volunteers in Israel. The FDA's Orphan Drug Designation program grants special status to drugs and biologics that are intended for use in rare diseases/disorders, defined as those that affect fewer than 200,000 people in the U.S. Orphan drug designation may qualify the sponsor for development incentives such as 7 years of U.S. market exclusivity, tax credits for qualified trials, the ability to apply for annual grant funding, clinical trial research design assistance and waiver of application fees associated with the new drug's approval under the Prescription Drug User Fee Act.