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.
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