iECURE, a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to its lead product candidate GTP-506 for the treatment of Ornithine Transcarbamylase (OTC) deficiency, a rare genetic condition that can lead to irreversible neurological impairment, seizures, coma and death, in a pediatric population.
“Receiving Rare Pediatric Disease Designation for GTP-506 for the treatment of OTC deficiency highlights the dire need for new treatment options for this devastating pediatric disease,” said Joe Truitt, Chief Executive Officer of iECURE. “GTP-506 is a potentially transformative therapy for babies born with OTC deficiency, and we expect to file an IND application with the FDA for our first-in-human clinical trial in mid-2023.”
The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application.