RARE Daily

FDA Grants Rare Pediatric Disease Designation to iECURE’s Gene Editing Treatment for OTC Deficiency

August 23, 2022

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to iECURE’s lead gene editing product candidate GTP-506 for the treatment of Ornithine transcarbamylase deficiency, a rare genetic condition that can lead to irreversible neurological impairment, seizures, coma and death, in a pediatric population.

Photo: Joe Truitt, CEO of iECURE

“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, CEO 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.”

Ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is a rare inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build up excessive levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma, and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. The only treatment for early onset severe OTC deficiency is a liver transplant. Currently available medical therapies do not correct the disease, and do not eliminate the risk of life-threatening symptoms or crises.

iECURE is a gene editing company, in which the University of Pennsylvania and James Wilson of Penn’s Gene Therapy Program hold equity interests focused, which is developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need.

iECURE’s approach to gene editing for its initial programs, including OTC deficiency, relies on the delivery of twin adeno-associated virus (AAV) capsids carrying different payloads. GTP-506 comprises two vectors, a gene editing vector that makes a cut in the well-characterized PCSK9 gene locus and a therapeutic donor vector that inserts the OTC gene to provide the desired genetic correction.

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. Recently, Marinus Pharmaceuticals sold its voucher to Novo Nordisk for $110 million.

Author: Rare Daily Staff

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