Release Date: 06-Sep-2021
US FDA has granted orphan drug designation for its investigational gene therapy product for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina. Efficacy and safety of the gene therapy are being evaluated in a Phase I/II clinical trial, which are currently enrolling patients.
The most common cause of blindness in children is Leber congenital amaurosis (LCA). This disease is caused by mutations in the GUCY2D gene and results in early and extreme vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA which affects roughly 20% of patients who live with this inherited retinal disease.
The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.