Eloxx pharmaceuticals receives orphan drug designation for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1) in US

Release Date: 29-Nov-2016

Eloxx Pharmaceuticals, a biotechnology firm focusing on developing and commercializing medicines and treatments for rare diseases, announces that Food and Drug Administration in US and European Medicine Agency (EMA) has granted orphan drug status to its leading product candidate, ELX-02 for the treatment of patients with Mucopolysacchardisis Type 1 (MPS 1).



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 Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.
The EC decision is based on a recommendation by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Orphan Drug Designation is granted for medicinal products that are intended for the diagnosis, prevention or treatment of rare diseases affecting less than 5 in 10,000 people in the European Union (EU). The Orphan Medicinal Product status of L19TNF entails a number of incentives, including 10-year market exclusivity once the product receives marketing authorisation.




For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

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