Axovant Gene Therapies Ltd (NASDAQ:AXGT) Receives Rare Pediatric Disease Designation For Its One-Time Gene Therapy AXO-AAV-GM2

Axovant Gene Therapies Ltd (NASDAQ:AXGT) recently announced that it was awarded rare disease designation for AXO-AAV-GM2, a gene therapy that it has been developing for treating Tay-Sachs and Sandhoff disease.

Axovant has been developing AXO-AAV-GM2 as a one-time gene therapy that is supposed to be administered directly into the central nervous system. The gene therapy was made to combat a type of GM2 gangliosidosis called Tay-Sachs and Sandhoff disease. The biopharma was pleased to announce that AXO-AAV-GM2 has been awarded Rare Pediatric Disease Designation by the U.S Food and Drug Administration (FDA).

The announcement is another big win for Axovant because the FDA previously awarded AXO-AAV-GM2 Orphan Drug Designation (ODD). The drug is also the first gene therapy given regulatory approval as a treatment viable for children diagnosed with Tay-Sachs disease.

“We are thrilled to bring AXO-AAV-GM2 one step closer to patients in need through this Rare Pediatric Disease designation,” stated Sean O’Bryan, the head of Regulatory Affairs & Quality at Axovant.

Axovant has so far observed consistent results from previous studies involving the gene therapy

Meanwhile, Axovant continues to develop gene therapy. The company is scheduled to run a clinical trial of AXO-AAV-GM2, which will feature a stage 1 dose-ranging study, as well as a stage 2 efficacy evaluation. The company reported favorable findings observed in previous studies of the gene therapy in which researchers observed significant disease modification potential in Tay-Sachs disease.

The previous study was an expanded access study in which two patients received AXO-AAV-GM2 successfully. The two patients demonstrated positive response to the treatment, and no adverse effects were observed. So far, gene therapy has been well-tolerated in clinical studies. The company is optimistic that the results of the ongoing and future trials will align with previous findings.

Axovant hopes to bring the one-time gene therapy to market if the findings will be favorable and as soon as it receives regulatory approval. Doing so will provide relief to patients suffering from Tay-Sachs and Sandhoff disease. It will be a vital therapy that could improve patients’ life expectancy, especially children suffering from the disease.