December 30, 2015 10:56am
For its gene therapy for the treatment of Mucopolysaccharidosis Type I (MPS I)
The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to RGX-111, the Company’s investigational gene therapy product candidate for the treatment of mucopolysaccharidosis Type I (MPS I).
The Bottom Line: The Rare Pediatric Disease Designation builds upon the Orphan Drug Designation granted earlier this year by the FDA to RGX-111 for MPS I, RGNX plans to file an IND application for RGX-111 for the treatment of MPS I in the first half of 2016.
The FDA defines 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 (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher which can be redeemed to obtain priority review for any subsequent marketing application.
RGNX closed at $17.61 and is DOWN -$0.11, still needs to file an IND.
MPS I is a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase (IDUA) gene. Over 1,000 individuals with MPS I are estimated to be born each year worldwide. Symptoms include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment. RGX-111 uses an AAV9 vector to deliver the IDUA gene to the central nervous system.
