Intellia Therapeutics Receives FDA RMAT Designation for Nexiguran Ziclumeran in Treating ATTR Amyloidosiswith Cardiomyopathy
Thursday, March 27, 2025
Intellia Therapeutics, Inc, a clinical-stage gene editing company focused on CRISPR-based therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for treating transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).
The RMAT designation, introduced under the 21st Century Cures Act, aims to accelerate the development and review of promising therapies intended to treat, modify, reverse, or cure serious or life-threatening diseases. This designation provides several advantages, including early interactions with the FDA to discuss surrogate or intermediate endpoints that may support accelerated approval and post-approval requirements. Additionally, it may lead to priority review of the therapy’s biologics licence application (BLA).
Nex-z has received RMAT designation from the U.S. FDA for both cardiomyopathy and polyneuropathy. It has also been granted Orphan Drug Designation by the U.S. FDA and the European Commission.
Developed using CRISPR/Cas9 gene-editing technology, nex-z is being investigated as a potential one-time treatment for transthyretin (ATTR) amyloidosis. The therapy is designed to inactivate the TTR gene responsible for producing the transthyretin (TTR) protein. Interim results from a Phase 1 clinical study indicated that a single dose of nex-z led to sustained and significant reductions in TTR protein levels. Intellia is leading its development and commercialisation in collaboration with Regeneron.
ATTR amyloidosis is a rare, progressive, and life-threatening disease. Hereditary ATTR (ATTRv) amyloidosis occurs due to mutations in the TTR gene, resulting in structurally abnormal TTR proteins that misfold and accumulate as amyloid deposits in the body. This can lead to serious complications affecting the heart, nerves, and digestive system. The disease manifests as polyneuropathy (ATTRv-PN), which can cause nerve damage, or cardiomyopathy (ATTRv-CM), which may lead to heart failure. In some cases, individuals without genetic mutations produce normal TTR proteins that become unstable over time, leading to amyloid deposits. This condition, known as wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart.
Approximately 50,000 people worldwide are estimated to have ATTRv amyloidosis, while between 200,000 and 500,000 individuals are affected by ATTRwt amyloidosis. There is currently no cure, and existing treatments focus on slowing the accumulation of misfolded TTR proteins.
Source: globenewswire.com
