The U.S. Food and Drug Administration (FDA) grants Regenerative Medicine Advanced Therapy (RMAT) designation to Intellia Therapeutics, Inc.‘s NTLA-2002 for the treatment of HAE. NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to inactivate the target gene, kallikrein B1 (KLKB1), to potentially prevent life-threatening swelling attacks in people with HAE.

“The RMAT designation is important recognition for our early clinical data. It indicates that a single dose of NTLA-2002 has the potential to address serious unmet medical need for people living with HAE,” says Intellia President and CEO John Leonard, M.D. “We look forward to continuing our productive dialogue with the FDA to accelerate the development of NTLA-2002, an investigational in vivo CRISPR-based therapy, with the goal of bringing forth a potentially transformative treatment to patients more quickly.”

The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, which are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA).

The RMAT is the third special regulatory designation received by Intellia for NTLA-2002. NTLA-2002 was also granted Orphan Drug Designation by the FDA and the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency.
(Source: Intellia)