Innorna’s IN013 Receives FDA Rare Pediatric Disease and Orphan Drug Designations for Wilson Disease
Boston, MA & Hong Kong (BUSINESS WIRE) – April 27, 2025 – Innorna, a clinical-stage biotechnology company pioneering mRNA therapeutics through its innovative lipid nanoparticle (LNP) delivery technology, today announced that the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to its investigational mRNA therapy, IN013, for the treatment of Wilson Disease (WD). This dual designation accelerates the clinical development of IN013, furthering Innorna’s commitment to providing transformative therapies for patients with WD.
About FDA Designations
The Rare Pediatric Disease Designation (RPDD) aims to encourage the development of therapies for serious or life-threatening diseases affecting fewer than 200,000 patients in the U.S., with the primary impact on individuals aged 18 years or younger. Upon approval of a product with RPDD, the sponsor may be eligible for a Priority Review Voucher (PRV), which can be used to expedite the FDA review of a subsequent drug application.
The Orphan Drug Designation (ODD) supports the development of therapies for rare diseases (affecting fewer than 200,000 people in the U.S.) by granting seven years of market exclusivity upon approval, providing tax credits for qualified clinical trials, and waiving certain FDA fees.
About Wilson Disease (WD) and IN013
Wilson Disease (WD), also known as Hepatolenticular Degeneration (HLD), is a rare and life-threatening autosomal recessive disorder caused by mutations in the ATP7B gene. These mutations lead to the toxic accumulation of copper in the liver, brain, kidneys, and other vital organs. The disease can manifest with a range of symptoms, including hepatic and neurological damage, ocular abnormalities, kidney injury, hemolysis, and skeletal complications. Current treatments often have limited efficacy and carry risks such as hepatorenal toxicity or the exacerbation of neurological symptoms.
IN013, developed utilizing Innorna’s advanced mRNA-LNP platform, is designed to address the underlying genetic cause of WD by restoring functional ATP7B protein levels. By effectively reducing the buildup of systemic copper, IN013 aims to alleviate multi-organ damage and deliver significant, disease-modifying benefits to patients.
Source:
https://www.drugdiscoveryonline.com/doc/innorna-announces-fda-pediatric-disease-orphan-drug-designations-wilson-disease-0001
About PackGene
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
