Home » Agilis’s AGIL-FA Gets Orphan Drug Designation
Agilis’s AGIL-FA Gets Orphan Drug Designation
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.
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