On November 25, 2024, Intellia Therapeutics announced that its CRISPR-based therapy, nexiguran ziclumeran (nex-z, NTLA-2001), received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is an in vivo gene editing therapy designed as a single-dose treatment to inactivate the TTR gene, thus preventing TTR protein production for the treatment of ATTR amyloidosis. Development and commercialization of nex-z is led by Intellia as part of a multi-target collaboration with Regeneron.
According to Intellia, the FDA’s decision follows promising interim Phase 1 results, showing rapid, deep, and durable TTR reduction after a single dose of nex-z. The RMAT designation, created under the 21st Century Cures Act, expedites development of therapies addressing serious conditions. The RMAT designation includes benefits, such as Early FDA engagement, use of surrogate or interfmediate endpoints for accelerated approval, and potential priority review of a Biologics License Application (BLA). Nex-z also holds Orphan Drug Designation from both the U.S. FDA and European Commission.