iECURE, a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ECUR-506, the company’s in vivo gene insertion program designed to treat neonatal onset Ornithine Transcarbamylase (OTC) deficiency.
The Fast Track designation is designed to facilitate the development and expedite the review of therapeutics to treat serious conditions that fill an unmet medical need. Therapeutics that receive Fast Track designation are eligible for more frequent meetings with and written communication from FDA to discuss the therapeutic’s development plan and ensure collection of appropriate data to support potential approval of the therapeutic. Provided relevant criteria are met, programs with Fast Track designation are eligible for accelerated approval and priority review as well.
“Receipt of Fast Track designation from the FDA is a validation of the severe unmet need for patients with neonatal onset OTC deficiency and a testament to the preclinical data generated to date for ECUR-506,” said Joe Truitt, Chief Executive Officer of iECURE. “The benefits of Fast Track designation may accelerate our ability to get ECUR-506 into physicians’ hands, which is incredibly important when every second counts for these babies.”
ECUR-506 previously received Rare Pediatric Disease and Orphan Drug designations from the FDA, and Orphan designation from the European Commission for the treatment of OTC deficiency.
iECURE’s OTC-HOPE study, a Phase 1/2 first-in-human study in newborn males with genetically confirmed neonatal onset OTC deficiency, is open for enrollment at the Great Ormond Street Hospital for Children in the United Kingdom. In addition, trial sites in the United States and Australia are activating and will be enrolling later this year.
The study is designed primarily to assess the safety and tolerability of ECUR-506 following intravenous administration of a single dose. Secondary objectives are to assess the pharmacokinetics and efficacy of ECUR-506. In addition, exploratory endpoints will assess disease-specific biologic markers, developmental milestones and quality of life.
