Lingyi Biotech's AAV Gene Therapy LY-M003 Receives FDA Orphan Drug and Rare Pediatric Disease Designations for Wilson’s Disease
Lingyi Biotech
Gene Therapy Drug Developer
August 7, 2024Lingyi Biotech Co., Ltd. (hereinafter referred to as "Lingyi Biotech") announced that its self-developed gene therapy drug LY-M003 injection has received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of Wilson’s disease (WD).This follows the FDA approval in mid-JulyPediatric Rare Disease Qualification (Rare Pediatric Disease Designation, RPDD）Later, the potential clinical value of LY-M003 injection was once again recognized by the FDA.
Orphan drug, also known as rare disease drug, refers to pharmaceuticals used for the prevention, treatment, and diagnosis of rare diseases. The orphan drug designation granted by the FDA applies to drugs and biologics for rare diseases with fewer than 200,000 patients annually in the United States. Drugs that obtain orphan drug designation are entitled to various incentives in the U.S., including expedited review for market approval, seven years of market exclusivity post-approval, waiver of NDA/BLA application fees, and tax benefits.
RPDDIt is a qualification designation for pediatric rare disease drugs targeting fewer than 200,000 patients in the U.S. and posing severe life-threatening risks to children under 18. Initiated by the FDA, the Rare Pediatric Disease Designation (RPDD) and Priority Voucher Program recognize the significant need for therapies addressing pediatric rare diseases, aiming to encourage the development of new treatments for these serious or life-threatening conditions. Under this program, sponsors will be eligible to receive a Priority Review Voucher (PRV) after the approval of a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease. The PRV can be used to accelerate the review of any subsequent product marketing application by six months or sold to a third party.
Lingyi Biotech Founder and CEO, Dr. Lin QingStated: "We are very pleased that LY-M003 Injection has been granted RPDD and ODD qualifications by the U.S. FDA, which isAnother important milestone in the company's development process.The company will continue to work closely with clinicians, researchers, and regulatory agencies to accelerate the clinical research and product development of LY-M003 injection, bringing safe and long-term effective therapeutic drugs to patients with Wilson's disease."
Hepatolenticular Degeneration (WD), also known as Wilson's Disease, is a rare autosomal recessive genetic metabolic disorder caused by mutations in the copper-transporting ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3), leading to impaired copper transport and resulting in the accumulation of copper ions in multiple organs such as the liver, brain, and kidneys, causing organ damage. The clinical manifestations of WD are complex, primarily involving liver and nervous system lesions, including abnormal liver function, hepatomegaly or splenomegaly, acute liver failure, neurodegeneration, neurological dysfunction, selective neural vulnerability, and executive dysfunction, among others. Involvement of other systems may also occur and could progressively worsen during the course of the disease.
LY-M003 Injection is an AAV gene therapy drug independently developed by Lingyi Biotech for the treatment of Wilson's disease. Using recombinant adeno-associated virus (rAAV) as a vector, it delivers the normal ATP7B gene into patients' hepatocytes through a single intravenous infusion, enabling long-term stable expression of functionally active ATP7B protein in hepatocytes. This restores copper metabolism in hepatocytes, reduces accumulated copper ions in the body, reactivates ceruloplasmin, and systematically improves systemic symptoms, thereby achieving the therapeutic goal for Wilson’s disease.
Lingyi Biotech was founded in February 2021 and is a global leader in the development of innovative drugs for monogenic inherited diseases. The company boasts a world-class team in gene therapy discovery, research, development, and industrialization, focusing on the R&D and commercialization of FIC gene therapy pipelines. With a commitment to original innovation, Lingyi Biotech concentrates on disease areas with urgent clinical needs such as metabolism, the central nervous system, and ophthalmology. Starting with key technologies like animal models of genetic diseases, the company aims to provide patients with one-time solutions according to the highest quality standards.
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