FDA Grants Orphan Drug Designation to Eloxx’s Enzyme Deficiency Drug

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.

The EMA grants Orphan Designation to medicines intended to treat, prevent or diagnose life threatening and debilitating disease, with a prevalence no greater than five in 10,000 in the EU, and where no satisfactory method of treatment, prevention or diagnosis exists, unless the proposed medicine offers a significant benefit to those with the condition. Following Orphan Designation, sponsors can access a number of incentives including protocol assistance, market exclusivity for a ten-year period following approval and potential fee reductions.

About Mucopolysacchardisis Type 1 (MPS I)

Mucopolysaccharidosis type I (MPS I), is a chronic, progressive genetic disorder caused by an   enzymatic deficiency of alpha-L-iduronidase (IDUA) which disrupts the glycosaminoglycan (GAG) catabolic pathway leading to an intra-lysosomal accumulation of substrates, heparan sulfate (HS) and dermatan sulfate (DS). The accumulation of HS and DS causes disruption in the movement of molecules inside the cell and the subsequent dysfunction of cells, tissues and organs. Globally, MPS I occurs in about 1 in every 100,000 births for the severe form and 1 in 500,000 for the attenuated form.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, is a biopharmaceutical company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations including: Cystic fibrosis, Duchene Muscular Dystrophy, Usher syndrome, Ataxia-telangiectasia, Beta thalassemia, Tay-Sachs Hurler syndrome and many others.

Eloxx Pharmaceuticals approach and knowhow allows moving rapidly from target drugs discovery program to clinical drug candidates. Building a robust pipeline of molecules designed for an array of nonsense genetic diseases. Translation of mRNA to a protein is the event where the cellular machinery, i.e. ribosome, ultimately dictates the quantity and timing for each protein to be produced. Thus, molecules that induce ribosomal readthrough overcome the nonsense mutations and allow production of a full-length functional protein. These molecules hold great therapeutic potential for the treatment of many genetic diseases.

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