LL schreef op 17 oktober 2023 08:38:
Intellia Therapeutics Receives Priority Medicines (PRIME) Designation From the European Medicines Agency for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary AngioedemaOctober 13, 2023 at 8:38 AM EDT
CAMBRIDGE, Mass., Oct. 13, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) 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.
“We are very pleased the EMA has granted PRIME designation to NTLA-2002. This designation provides valuable regulatory benefits and highlights the potential of our novel in vivo gene editing candidate to address an unmet medical need for people living with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We look forward to continuing to work closely with the EMA and regulatory agencies around the world to bring this innovative therapy to patients as quickly as possible.”
PRIME designation is granted by the EMA to drug candidates that may offer a major therapeutic advantage over existing treatments or that benefit patients without treatment options. The PRIME designation was created by the EMA to provide early and proactive support to developers of promising medicines to optimize their development plans and speed up evaluation. The goal is to help patients benefit as early as possible from innovative new therapies that have demonstrated potential to significantly address unmet medical need.
PRIME designation was granted based on positive interim data from the Phase 1 portion of the ongoing Phase 1/2 clinical trial of NTLA-2002 in patients with HAE. In June, Intellia announced that across all 10 patients dosed in the Phase 1, a 95% mean reduction in monthly attack rate was observed after a single dose of NTLA-2002 through the latest follow-up. The median duration of follow-up was 9.0 months (range of 5.6 – 14.1 months). At all three dose levels evaluated, NTLA-2002 has been well tolerated, and the majority of adverse events were mild in severity.
PRIME is the fourth specialty regulatory designation Intellia has received for NTLA-2002. NTLA-2002 was also granted Orphan Drug Designation and RMAT Designation by the U.S. Food and Drug Administration (FDA), as well as the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA).
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ir.intelliatx.com/news-releases/news-...