Eloxx pharmaceuticals announces that its leading drug candidate has been approved as an orphan drug.
Release Date: 02-Dec-2016
Eloxx pharmaceuticals announces that its leading drug candidate has been approved as an orphan drug for the treatment of patients with novel mucopolysaccharidosis Tx type 1 (MPS I) by the US Food and Drug Administration (FDA). ELX-02 is a synthetic design of aminoglycoside enhanced as translational read through drug (TRID) for the treatment of genetic diseases caused by nonsense mutations. Currently, the drug is undergoing its phase 1 (first in Human) clinical trial.
Mucopolysaccharidosis type I (MPS I) is a chronic, progressive genetic disorder caused by an enzymatic deficiency of alpha-L-iduronidase (IDUA) which interrupts the glycosaminoglycan (GAG) catabolic pathway leading to an intra-lysosomal accumulation of substrates, heparan sulfate (HS) and dermatan sulfate (DS).
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.
For Orphan Drug Clinical Insight Reports Contact: firstname.lastname@example.org
Need custom market research solution? We can help you with that too.
Kuick Research is a market research and analytics company that provides targeted information for critical decisions at business, product and
service levels. We are quick, predictive and known by the recommendations we have made in the past. Our result-oriented research methodology offers
understanding of multiple issues in a short period of time and gives us the capability to keep you full with loads of practical ideas.