EMA grants PRIME designation to NTLA-2002 gene-editing therapy

Hereditary angioedema treatment has begun dosing in a Phase 2 trial

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by Steve Bryson, PhD |

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The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to Intellia Therapeutics’ NTLA-2002, an experimental gene-editing therapy to prevent swelling attacks in people with hereditary angioedema (HAE).

A PRIME designation supports the development of experimental therapies that address unmet medical needs. Eligibility for this status is based on early clinical data demonstrating that the medicine may provide advantages over existing therapies or benefit patients who have no other treatment options.

The designation also allows for early and proactive support by the EMA to the developers of these medicines to help optimize their clinical plans and speed up the review process.

“We are very pleased the EMA has granted PRIME designation to NTLA-2002,” John Leonard, MD, Intellia’s CEO and president, said in a company press release. “This designation provides valuable regulatory benefits and highlights the potential of our novel in vivo gene editing candidate to address an unmet medical need for people living with hereditary angioedema.

“We look forward to continuing to work closely with the EMA and regulatory agencies around the world to bring this innovative therapy to patients as quickly as possible.”

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CRISPR/Cas-9 gene-editing used in therapy

Swelling attacks associated with HAE are driven by elevated levels of a pro-inflammatory molecule called bradykinin, whose production is, in turn, controlled by the enzyme kallikrein.

NTLA-2002 uses the CRISPR/Cas-9 gene-editing system to disrupt the KLKB1 gene, which encodes a kallikrein’s precursor protein called prekallikrein. As such, NTLA-2002 is expected to reduce kallikrein activity, lower bradykinin levels, and prevent swelling attacks.

The PRIME designation was supported by interim data from the Phase 1 portion of an ongoing Phase 1/2 clinical trial (NCT05120830) evaluating NTLA-2002 in HAE patients. Results from this part of the study showed that months after a single intravenous (into-the-vein) infusion of NTLA-2002 at doses of 25, 50, or 75 mg, 9 out of 10 people with HAE were free from swelling attacks.

Across all 10 patients, the mean monthly attack rate decreased by 95% at the latest follow-up, which ranged from about six to 14 months. According to Intellia, all three doses of NTLA-2002 were well tolerated, and most adverse events were mild in severity.

The Phase 2 portion of the study, which has already begun dosing, is testing two doses of NTLA-2002 against a placebo. Its main goal is to assess the treatment’s ability to lower attack rates after a period of 16 weeks.

The company also plans to launch a Phase 3 trial in 2024, including sites in the U.S., to generate the data needed to support a future application seeking NTLA-2002’s approval. Before this trial, Intellia will provide preclinical data about including women of childbearing age in NTLA-2002’s clinical program.

Previously, the U.S. Food and Drug Administration (FDA) granted NTLA-2002 the designations of  regenerative medicine advanced therapy and orphan drug. These are meant to encourage and speed the development of therapeutics for rare and serious conditions. The U.K. Medicines and Healthcare products Regulatory Agency also has  granted the investigational therapy the innovation passport.