NEW YORK, Oct. 09, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ:AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, today announced that it has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for AXO-AAV-GM1, an AAV9-based gene therapy delivered via a single intravenous administration that is in Phase 1/2 development for GM1 gangliosidosis. In addition to the Rare Pediatric Disease designation, AXO-AAV-GM1 has Orphan Drug designation and is the only gene therapy in clinical development for both infantile (Type I) and juvenile (Type II) GM1 gangliosidosis.
“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,” said Sean O’Bryan, Senior Vice President, Regulatory Affairs & Quality. “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.”
Axovant is on-track to report 6-month Stage 1 data from the low dose juvenile cohort (Type II) by the fourth quarter of 2020, and expects to initiate the high dose cohort which includes infantile (Type I) and juvenile (Type II) patients in the second half of 2020.
GM1 Gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the β-galactosidase enzyme. Currently, there are no approved treatment options for GM1 Gangliosidosis.
The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the United States.