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Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema
CAMBRIDGE, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative single-dose therapeutics leveraging CRISPR-based technologies, today announced that the European Commission (EC) has granted orphan drug designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to prevent potentially life-threatening swelling attacks in people with HAE. “The European Union orphan drug designation for NTLA-2002 represents another important milestone for Intellia as we continue to make rapid progress in the development of a novel, potential one-time treatment for people with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are on track to complete enrollment of the Phase 2 portion of the study in the coming weeks, which will bring us one step closer to our goal of delivering a potentially life-changing treatment for people who suffer from this serious and debilitating disease.” Orphan drug designation in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for orphan drug designation, a candidate therapy must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that occurs in not more than five in 10,000 people in the EU. The designation provides regulatory, financial and commercial incentives to develop therapies for rare diseases where there are either no satisfactory treatment options or significant benefit to those affected by the disease. Intellia has received five special regulatory designations for NTLA-2002. NTLA-2002 was also granted Orphan Drug Designation and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), as well as PRIME designation by the EMA. About the NTLA-2002 Clinical ProgramIntellia’s global Phase 1/2 study is evaluating the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). This includes the measure...