Innorna Announces FDA Rare Pediatric Disease and Orphan Drug Designations Granted to IN013 for Treatment of Wilson Disease
BOSTON & HONG KONG--(BUSINESS WIRE)-- Innorna, a clinical-stage biotechnology company revolutionizing mRNA therapeutics with its innovative lipid nanoparticle (LNP) delivery technology, today announced 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 treating Wilson Disease (WD). This dual achievement accelerates the clinical development of IN013, advancing Innorna's mission to deliver transformative therapies for WD patients.
Innorna receives dual FDA designations for IN013, marking a critical milestone in developing an mRNA therapy for Wilson Disease.
About FDA Designations
The Rare Pediatric Disease Designation (RPDD) incentivizes therapies for serious or life-threatening diseases affecting fewer than 200,000 U.S. patients, primarily those aged 18 or younger. Sponsors may qualify for a Priority Review Voucher (PRV) upon approval to expedite FDA review of a subsequent drug application.
The Orphan Drug Designation (ODD) supports therapies targeting rare diseases (affecting fewer than 200,000 U.S. patients) by providing seven years of market exclusivity, tax credits for clinical trials, and waiver of certain FDA fees.
About Wilson Disease (WD) and IN013
Wilson Disease (WD), or Hepatolenticular Degeneration (HLD), is a rare and life-threatening autosomal recessive disorder caused by mutations in the ATP7B gene, leading to toxic copper accumulation in the liver, brain, kidneys, and other organs. Symptoms include hepatic and neurological damage, ocular abnormalities, kidney injury, hemolysis, and skeletal complications. Current treatments offer limited efficacy and carry risks such as hepatorenal toxicity or exacerbated neurological symptoms.
IN013, developed using Innorna's mRNA-LNP platform, targets the root cause of WD by restoring functional ATP7B protein levels. By reducing systemic copper buildup, IN013 aims to alleviate multi-organ damage and deliver disease-modifying benefits.
About Innorna
Founded in 2019, Innorna is committed to developing state-of-the-art LNP delivery systems and RNA therapies to address unmet medical needs. Its proprietary Diversity-Oriented Lipid Library —with over 5,000 ionizable lipids—enables breakthroughs in mRNA vaccines, gene editing, and cell therapies.
With its innovative mRNA-LNP platform, Innorna has built a robust pipeline targeting infectious diseases, rare genetic disorders, and cancer immunotherapy. Beyond its internal R&D efforts, the company collaborates with global-leading biotechnology partners to expand the reach of its transformative technology.
Innorna's achievements have earned recognition as one of MIT Technology Review's Global Top 50 Smartest Companies and Fortune China's Most Socially Influential Startups. Guided by INNOVATION, INTEGRITY, EFFICIENCY, and OPENNESS, Innorna drives its mission to revolutionize mRNA applications and make a global impact.
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Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella's clinical development plans and related anticipated development milestones, Palvella's cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella's programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella's product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella's product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella's limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella's current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. 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