Lingyi Biotech's AAV Gene Therapy LY-M001 Receives FDA Orphan Drug Designation for Gaucher Disease

Oct 14, 2023 19:38 CST Updated 19:38

Lingyi Biotech

Gene Therapy Drug Developer

Recently, LY-M001 Injection, the first candidate drug independently developed by Lingyi Biotech Co., Ltd. (Hangzhou), has been granted Orphan-drug Designation (ODD) by the Office of Orphan Products Development of the U.S. FDA for the treatment of Gaucher's disease.

The pathogenesis of Gaucher's disease is due toGBA1Gene mutations lead to a lack or reduction in the activity of glucocerebrosidase in the body, causing its substrate, glucocerebroside, to accumulate in the lysosomes of macrophages in the liver, spleen, bones, lungs, and even the brain, forming typical storage cells known as "Gaucher cells." This accumulation results in lesions in bodily tissues and organs, which progressively worsen. The prevalence of Gaucher disease varies across different regions globally. According to a statistical analysis, the number of affected individuals per 100,000 people worldwide ranges from 0.7 to 1.75, making it one of the most common lysosomal storage disorders globally.

LY-M001 Injection is a gene therapy product using recombinant adeno-associated virus (rAAV) as a vector. After a single intravenous infusion, it specifically transduces the target organ, the liver, and enables long-term stable expression of the modified glucocerebrosidase in hepatocytes. Glucocerebrosidase can be secreted into the bloodstream and absorbed by other organs, degrading the harmful glycolipid metabolites accumulated in various organs of the patient, thereby effectively treating Gaucher's disease.

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 granted orphan drug designation can enjoy incentives such as expedited review for marketing applications in the U.S., seven years of market exclusivity post-approval in the U.S., waiver of NDA/BLA application fees, and tax benefits. Gaucher's disease is also included in China's first list of rare diseases.

The granting of FDA Orphan Drug Designation for LY-M001 Injection in the treatment of Gaucher disease marks an important milestone in the company's development, and will play a positive role in accelerating the advancement of clinical trials and registration for LY-M001 Injection in both China and the United States.

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