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BioWorld - Tuesday, June 2, 2026
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Home » IN-016 granted orphan status for progressive familial intrahepatic cholestasis therapy
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Genetic/congenital

IN-016 granted orphan status for progressive familial intrahepatic cholestasis therapy

Aug. 2, 2024
Innorna Co. Ltd.’s IN-016 has received orphan drug status from the U.S. FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC). Earlier in July, IN-016 had been granted Rare Pediatric Disease Designation. PFIC is a group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver.
BioWorld Science Regulatory RNA Gastrointestinal Genetic/congenital FDA IND Orphan drug

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