The FDA has issued a rare pediatric disease designation to a gene therapy for the treatment of children with GM1 gangliosidosis, according to the drug’s manufacturer.
GM1 gangliosidosis, also known as GM1, is a rare autosomal recessive disease that develops from a mutation in the GLB1 gene encoding for the lysosomal acid beta-balactosidase enzyme, causing a deficiency that leads to accumulation of GM1-ganlioside in cells.
Children with GM1 often present with neurological impairment, including rapid loss of motor, cognitive and behavioral skills, leading to premature death. The disease most commonly affects very young children and currently no modifying treatment is available.
LYS-GM101 (Lysogene) is a gene therapy candidate for the treatment of GM1 that may help replace the defective GLB1 gene in the cells of affected children. This therapy is intended to encourage functional enzyme production and prevent further neurological damage caused by GM1.
“This rare pediatric disease designation for LYS-GM101 is Lysogene’s second designation after the LYS-SAF302 designation for the treatment of MPS IIIA [also known as Sanfilippo syndrome],” Karen Aiach, founder and chief executive officer of Lysogene, said in a press release. “It is an important validation of Lysogene’s work in the rare disease space and is indicative of our commitment to achieving a meaningful impact on the lives of the patients affected by GM1 and their families.”
LYS-GM101 will advance into upcoming phase 1/2 clinical trials in future months.
The FDA has defined a “rare pediatric disease” as a disease that affects fewer than 200,000 individuals in the U.S., primarily aged from birth to 18 years. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a rare pediatric disease may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product, and may be sold or transferred.