Lysogene announced that the U.S. FDA has granted Fast Track Designation to its LYS-SAF302 program for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA). LYS-SAF302, a second-generation gene therapy, is designed to deliver a functional copy of the SGSH (N-sulfoglucosamine sulfohydrolase) gene to the brain through a one-time direct-to-CNS administration.
The Fast Track program facilitates the development and accelerates the review of new drugs for serious conditions, which have the potential to address unmet medical needs. A product that receives Fast Track designation is eligible for more frequent interactions with FDA, potential eligibility for accelerated approval, priority review, and rolling Biologics License Application (BLA) review.
An international phase 2/3 trial, AAVance, is currently underway. The open-label, single-arm, multicenter study of LYSSAF302 for the treatment of MPS IIIA is designed to include 20 patients with the severe classical form of MPS IIIA. Currently, a total of 17 patients have been treated out of the total of 20. The primary objective is to assess the drug efficacy in improving the neurodevelopmental status of patients after 24 months, compared to the expected evolution based on natural history data. Safety, tolerability, effect on behavior, sleep and quality of life will also be collected as secondary endpoints
LYS-SAF302 has previously received Orphan Drug Designations for the treatment of MPS IIIA in the European Union in 2014 and in the US in 2015, as well as Rare Pediatric Disease Designation in the US.
LYS-SAF302 is an AAV-mediated gene therapy, the goal of which is to replace the faulty SGSH gene with a healthy copy of the gene. LYS-SAF302 employs the AAVrh10 virus, chosen for its ability to target the central nervous system.
About MPS IIIA
MPS IIIA is a rare inherited neurodegenerative lysosomal storage disorder affecting approximately 1 in 100,000 newborns. Inherited in an autosomal recessive pattern, it is characterized by intractable behavioral problems and developmental regression resulting in early death. It is caused by mutations in the SGSH gene, which encodes an enzyme called Heparan-N-sulfamidase necessary for heparan sulfate (HS) recycling in cells. The disrupted lysosomal degradation and resulting storage of HS and glycolipids such as gangliosides leads to severe neurodegeneration. There are currently no treatment options for patients.