Shenzhen-based Innorna Co., Ltd has announced that it has received Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for its proprietary mRNA drug, IN015, developed for the treatment of progressive familial intrahepatic cholestasis (PFIC).
Understanding PFIC and the Impact of IN015
Progressive familial intrahepatic cholestasis (PFIC) is a rare group of heterogeneous autosomal recessive genetic diseases resulting from gene mutations that cause bile exclusion disorders and intrahepatic bile stasis. Patients with PFIC typically exhibit symptoms such as jaundice, hepatomegaly, itching, splenomegaly, and diarrhea. The condition is progressive, potentially leading to liver injury, fibrosis, and cirrhosis, which can significantly increase the mortality rate. Currently, there is a dearth of effective treatment options for PFIC, with clinical practice mostly relying on supportive treatment.
IN015 is an mRNA drug designed to restore defective proteins and normalize bile excretion, thereby alleviating symptoms in PFIC patients and fundamentally addressing the genetic cause of progressive familial intrahepatic cholestasis. Earlier this year, Innorna’s IN016, another mRNA drug targeting PFIC, was also granted RPDD in the US. These two drugs, targeting different functional proteins, are anticipated to offer more comprehensive therapeutic effects for PFIC patients.- Flcube.com
