US FDA grants Rare Pediatric Disease Designation to Axovant’s AAV9-based gene therapy, AXO-AAV-GM1 for GM1 gangliosidosis
Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies for neurological diseases, announced that it has received Rare Pediatric Disease Designation from the US 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 ß-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.
AXO-AAV-GM1 is an investigational gene therapy that delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring ß-galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1’s ability to improve ß-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.
In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.
Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases.
