RARE Daily

FDA Grants RMAT Designation to Intellia’s Experimental Treatment for Hereditary Angioedema

March 21, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to Intellia Therapeutics’ NTLA-2002 for the treatment of hereditary angioedema.

Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE, and current treatment options often include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.

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 hereditary angioedema,” said John Leonard, president and CEO of Intellia.

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.

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.

Intellia’s multi-national phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). This includes the measurement of plasma kallikrein protein and activity, as well as HAE attack rate. The phase 1 portion of the study is an open-label, single-ascending dose design used to identify two dose levels of NTLA-2002 for further evaluation in the phase 2, randomized, placebo-controlled portion of the study. This phase 1/2 study will identify the dose of NTLA-2002 for use in future studies. In 2022, Intellia reported positive interim results from the phase 1 study demonstrating deep, dose-dependent reductions in plasma kallikrein and robust reductions in patient HAE attacks. Patient screening in the phase 2 portion of the study is ongoing.

Photo: John Leonard, president and CEO of Intellia

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