Chondrial Therapeutics, Inc., an emerging biotechnology company focused on the treatment of rare mitochondrial diseases, announced today that the US Food and Drug Administration (FDA) has granted orphan drug designation to its lead investigational drug candidate, CTI-1601, being developed for the treatment of Friedreich's Ataxia.
Orphan drug designation is granted by the FDA Office of Orphan Products Development to drugs being developed for safe and effective treatment of diseases that affect fewer than 200,000 people in the U.S. For a drug to qualify for orphan drug designation, FDA must determine there is a medically plausible basis for the use of the drug for the rare disease or condition.
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