Shenzhen-based Innorna Co., Ltd, a leading platform company specializing in mRNA technology and LNP (lipid nanoparticle) delivery technology, has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its investigational mRNA drug, IN015. The designation is for the treatment of progressive familial intrahepatic cholestasis (PFIC).
IN015: A Potential Treatment for PFIC
IN015 is designed to address the root cause of progressive familial intrahepatic cholestasis by restoring defective proteins and normalizing bile excretion, thereby alleviating symptoms in PFIC patients. This innovative approach aims to fundamentally treat PFIC, a condition caused by genetic mutations. The drug’s mechanism of action highlights the potential of mRNA technology in addressing genetic diseases.
Building on Previous FDA Designation
The receipt of ODD for IN015 follows the previous acquisition of a Rare Pediatric Disease Designation (RPDD) from the FDA. These designations underscore the potential impact of IN015 in treating rare diseases and highlight Innorna’s commitment to developing novel therapies for conditions with significant unmet medical needs.-Fineline Info & Tech
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