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European Commission Grants Orphan Designation for iECURE’s Lead Product Candidate GTP-506 for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency
PHILADELPHIA--(BUSINESS WIRE)-- iECURE, a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing
About this update from Precision Biosciences, Inc.
[{"type":"text","content":" PHILADELPHIA--(BUSINESS WIRE)--\niECURE, 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, announced today that the European Commission granted orphan designation for the company’s lead product candidate GTP-506 for the treatment of Ornithine Transcarbamylase (OTC) deficiency.\n\nGTP-506 previously received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for the treatment of OTC deficiency.\n\n“Securing Orphan Drug designation in both the U.S. and the EU highlights the work we have done to demonstrate how our in vivo gene editing approach has the potential to provide clinical benefit to patients who are in desperate need for treatments for OTC deficiency,” said Joe Truitt, Chief Executive Officer of iECURE. “Without programs like Orphan designation, thousands of patients with a myriad of rare diseases would have no medicines to treat their disorders, leading to uncontrolled symptoms and possible death. Achieving this designation brings us one step closer to providing a potentially transformative therapy to children living with this devastating disease.”\n\nOrphan designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. The European Commission grants orphan status for products intended for the treatment, prevention, or diagnosis of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU, and where there is no satisfactory method of treating, preventing, or diagnosing such condition authorized for marketing in the EU or if such a method exists, the product represents a significant benefit to those affected by the condition. Orphan designation provides companies with certain benefits and incentives in the EU, including clinical protocol assistance, possible waivers or reductions in regulatory fees and ten years of market exclusivity after approval.\n\nAbout OTC Deficiency\n\nOTC deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for the detoxification of ammonia....