This article is more than 5 years old. Information may no longer be current.
FDA grants rare pediatric disease label to investigational drug for NPC
Click Here to Manage Email Alerts
The FDA has issued rare pediatric disease designation to an investigational gene therapy product candidate for the treatment of children with Niemann-Pick Type C1 disease, a progressive, irreversible, chronically debilitating and fatal genetic disease, according to the drug’s manufacturer.
Niemann-Pick Type C1 disease (NPC) is caused by a lipid transportation defect within the cell that incurs increased lipids in the brain, liver and spleen. NPC usually affects children aged between 4 and 7 years and there is estimated to be only 150,000 cases globally.
VTS-270 (Vtesse Inc.) is a well-characterized mixture of 2-Hydroxypropyl-beta-cyclodextrin that carries a specific compositional fingerprint different from other 2-Hydroxypropl-beta-cyclodextin mixtures intended to slow progression and treat NPC. Vtesse Inc., in collaboration with the NIH’s National Center for Advancing Translational Sciences and Eunice Kennedy Shriver National Institute of Child Health and Human Development, conducted the preclinical research and initiated therapy development for VTS-270.
“We recognize the considerable collaboration and efforts to advance the clinical development and regulatory processes in the rare disease space, and, above all, we continue to thank all of the parents who have supported the development of VTS-270,” Ben Machielse, Drs., President and CEO of Vtesse, said in a press release.
VTS-270 is currently under investigation in a pivotal Phase 2b/3 clinical trial in NPC. Vtesse is enrolling patients in the U.S., the U.K., France, Germany, Spain, Turkey and Australia.
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 infancy 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.