www.fdanews.com/articles/177831-agiliss-agil-fa-gets-orphan-drug-designation
Agilis’s AGIL-FA Gets Orphan Drug Designation
August 4, 2016
The FDA has granted orphan drug designation for Agilis’s gene therapy candidate AGIL-FA.
The product is being developed to treat the degenerative neuromuscular disorder Friedreich’s ataxia.
Agilis says it is the first company to receive orphan drug designation from the FDA for a gene therapy to treat the condition.