Innorna, a clinical-stage biotechnology company focused on innovative mRNA therapies, announced that the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to IN013, its investigational therapy targeting Wilson Disease (WD).
Wilson Disease is a rare genetic disorder caused by mutations in the ATP7B gene, which impairs the body’s ability to eliminate excess copper. This leads to dangerous copper buildup in vital organs such as the liver, brain and corneas, causing life-threatening liver disease, neurological symptoms, psychiatric issues and even fatal complications if untreated. Early diagnosis and treatment are crucial to prevent irreversible organ damage.
IN013 is a liver-targeted mRNA therapeutic encapsulated in proprietary lipid nanoparticles (LNPs) developed by Innorna. It is designed to restore normal ATP7B protein function, addressing the root cause of Wilson Disease rather than just managing symptoms. Preclinical studies have shown encouraging data regarding the therapy’s ability to correct copper metabolism and improve survival outcomes.
The Rare Pediatric Disease Designation not only highlights the unmet need for pediatric patients but also makes Innorna eligible to receive a Priority Review Voucher (PRV) upon potential FDA approval, expediting the review of future therapies. Meanwhile, the Orphan Drug Designation offers critical incentives, including seven years of market exclusivity in the U.S., tax credits for clinical research costs, and waived FDA user fees.
IN013 is currently moving toward clinical trials. The FDA’s recognition through these designations is expected to accelerate its development, bringing hope to patients with few existing treatment options.
“Receiving both RPDD and ODD for IN013 is a major milestone in our mission to transform the lives of patients with rare genetic diseases,” said Dr. Xiaobin He, CEO of Innorna.
Wilson Disease affects approximately one in every 30,000 people worldwide, and despite available treatments like copper chelation therapy and liver transplantation, there remains a significant need for therapies that correct the underlying genetic defect.
Disclaimer: This article is for informational purposes only and does not constitute medical advice. IN013 is an investigational therapy and has not yet been approved for general clinical use.
