RARE Daily

FDA Grants Fast Track Designation for Lysogene’s Gene Therapy for Sanfilippo Syndrome Type A

February 26, 2020

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation for French biotech Lysogene’s experimental gene therapy, LYS-SAF302, for the treatment of lysosomal storage disorder Sanfilippo syndrome type A.

Sanfilippo syndrome, or MPS IIIA, is a rare inherited neurodegenerative disease affecting approximately 1 in 100,000 newborns. 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.

LYS-SAF302 is a second-generation gene therapy designed to deliver a functional copy of the SGSH gene to the brain through a one-time direct-to-central nervous system administration, and is being investigated in an international late-stage clinical trial. So far 17 patients out of 20 have been dosed with the therapy. The primary objective of the study 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. Lysogene has also set up the sub study to collect supportive video outcomes in the home environment.

“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,” said Karen Aiach, CEO of Lysogene.

The Fast Track program facilitates the development and accelerates the review of new drugs for serious

conditions that 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 review.

LYS-SAF302 has Orphan Drug designations in both the United States and Europe, as well as Rare Pediatric Disease designation in the United States. In October 2018, Lysogene licenses ex-European commercial rights to the gene therapy to Sarepta Therapeutics for up to $150 million.


Photo: Karen Aiach, CEO of Lysogene

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