AXGT - Axovant nabs Rare Pediatric Disease tag for AXO-AAV-GM2 in rare neurodegenerative disorder

The FDA has granted Rare Pediatric Disease Designation to Axovant Gene Therapies (AXGT) for AXO-AAV-GM2, a gene therapy delivered directly to the central nervous system for GM2 gangliosidosis, also known as Tay-Sachs and Sandhoff disease, which is a rare and fatal pediatric neurodegenerative lysosomal storage disorder resulting from deficiencies in beta-hexosaminidase.Rare Pediatric Disease designation provides for the issuance of a priority review voucher following FDA approval. The voucher can be used for accelerated approval of a future application or it can be sold to a third party. The company expects to evaluate AXO-AAV-GM2 in a registrational clinical trial which consists of a Stage 1 dose-ranging study and a Stage 2 efficacy study.Shares up 4% premarket.Previously: Axovant Gene Therapies candidate nabs Rare Pediatric Disease tag for nerve cell disorder (Oct. 9)

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Axovant nabs Rare Pediatric Disease tag for AXO-AAV-GM2 in rare neurodegenerative disorder