Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA
Lysogene (Paris:LYS) (FR0013233475 – LYS), a pioneering Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (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, and is being investigated in the international Phase 2/3 clinical trial AAVance (NCT03612869).
Karen Aiach, Founder and Chief Executive Officer said: “MPS IIIA is a lethal neurological disease with debilitating symptoms for which there is currently no treatment. The FDA’s recognition of LYS-SAF302’s potential to improve neurocognitive deficits in children with MPS IIIA represents an important achievement for Lysogene and the patient community.” Karen Aiach added: “We are also pleased to announce the treatment of the 17th patient with LYS-SAF302, which once again demonstrates our capacity to execute with quality and speed. LYS-SAF302 is the flagship of our differentiated direct-to-CNS gene therapy platform, which has been validated not only by our partner Sarepta, but also by the scientific community. We are fully dedicated to continue the full speed development of LYS-SAF302, as well as all the other programs in our pipeline.”
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. The purpose is to expedite the availability of new treatment options for patients. 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.
“This Fast Track designation demonstrates the regulators’ sustained interest in Lysogene’s cutting edge in vivo gene therapy program. 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,” added Marie Deneux, Chief Regulatory Officer. “In the complex field of gene therapy for neurodegenerative diseases, a continued communication with FDA is essential.”