Regenxbio Scores Rare Pediatric Disease Designation for Gene Therapy

Regenxbio has won a FDA rare pediatric disease designation for its investigational gene therapy candidate for the treatment of mucopolysaccharidosis type I.

RGX-111 uses an adeno-associated virus vector to deliver the human IDUA gene to the central nervous system, which MPS I lacks. The disease strikes about 1 in 100,000 newborns.

Regenxbio has said it plans to file an IND application with the FDA in the first half of the year.

Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an NDA or BLA approval may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application.