Axovant gets FDA rare pediatric designation for gangliosidosis gene therapy

Axovant Logo

Axovant Gene Therapies (NASDAQ:AXGT) received FDA orphan drug designation for AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.

GM1 gangliosidosis is a progressive and fatal 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. The drug candidate is currently in a Phase 1/2 study for both infantile (Type I) and juvenile (Type II) forms of the disease, with six-month data from the low-dose cohort expected by the end of 2020. 

“Receiving rare pediatric disease designation in addition to the previously granted orphan drug designation for AXO-AAV-GM1 highlights the importance of expediting a therapy for GM1 gangliosidosis, a progressive, fatal, pediatric disease with no approved treatment options,” Sean O’Bryan, Axovant’s SVP of regulatory affairs and quality, said in a statement.

“AXO-AAV-GM1 is the first gene therapy to enter clinical trials for GM1 gangliosidosis and has the potential to provide meaningful clinical benefit to both Type I and Type II patients,” he added. 

Sign up for the BioTuesdays weekly newsletter

Get the latest news on the healthcare industry’s corporate and clinical developments, executive moves, and market updates. Every Tuesday, in your inbox.