Axovant Gene Therapies (NASDAQ:AXGT) received FDA orphan drug designation for AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.
GM1 gangliosidosis is a rare genetic disease where mutations in the GLB1 gene impair production of beta-galactosidase, causing lipids to accumulate in cells of the nervous system.
AXO-AAV-GM1 is designed to deliver a functional copy of the GLB1 gene to restore normal beta-galactosidase production. Axovant is enrolling patients in the first part of a Phase 1/2 study, with six-month data expected in mid-2020.
“There are currently no FDA approved therapies for patients with GM1 gangliosidosis, and the orphan drug designation underscores the significant unmet medical need,” Dr. Gavin Corcoran, chief R&D officer, said in a statement.
