FDA Awards Lysogene Orphan Drug, Rare Pediatric Disease Designations

December 3, 2015

Paris-based Lysogene received orphan drug and rare pediatric disease designations from the FDA for its LYS-SAF-302 gene therapy candidate for treating patients with Sanfilippo Type A, also known as mucopolysaccharidosis type IIIA.

The therapy has the potential to replace the defective gene in the cells of Sanfilippo Type A patients, thus allowing for the production of the enzyme and the prevention of progressive neurological damage, the company says.

The adeno-associated virus gene therapy recently completed a Phase 1/2 trial, and a Phase 2/3 trial is expected to begin shortly.