The U.S. Food & Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), according to a recent press release from Intellia Therapeutics.
“The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease,” Intellia wrote.
RMAT designation provides several benefits including more frequent engagement with the FDA and the potential for accelerated approval. Drugs with this designation are also eligible to apply for priority review of their biologics license application, which may expedite market availability.
Nex-Z is a CRISPR/Cas9 gene editing therapeutic that works by inactivating the TTR gene, which encodes the transthyretin protein. By limiting the production of transthyretin, nex-z helps prevent the buildup of misfolded protein in patients with ATTR-CM.
Read more about ATTR-CM therapies
RMAT designation was provided based on promising results from a Phase 1 clinical trial. The study, which included patients with ATTR-CM and hereditary transthyretin amyloid polyneuropathy (hATTR-PN), found that nex-z resulted in sustained and prolonged reduction of transthyretin levels.
The drug previously received Orphan Drug Designation from the FDA and the European Commission. The addition of RMAT designation not only provides additional regulatory advantages but also opens new avenues for priority review.
Several drugs have been approved for the treatment of ATTR-CM, including vutrisiran, tafamidis and acoramidis. However, all of these drugs require repeated dosing to maintain their efficacy. If approved, nex-z will be the first treatment for ATTR-CM that only requires a single dose.
Intellia Therapeutics is collaborating with Regeneron Pharmaceuticals to develop nex-z.
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