NTLA-2002 earns orphan drug designation in Europe
Potential gene-editing treatment targets hereditary angioedema
The designation is given to therapies with the potential to improve medical care for people with serious conditions that affect up to five of every 10,000 people in Europe. It confers certain regulatory, financial, and commercial incentives to encourage the development of treatments for rare disorders where there might not be a strong economic motivation for companies.
If ultimately approved by the EC, orphan drugs are guaranteed a decade free from competition with generics and other formulations.
“The European Union orphan drug designation for NTLA-2002 represents another important milestone for Intellia as we continue to make rapid progress in the development of a novel, potential one-time treatment for people with hereditary angioedema,” John Leonard, MD, president and CEO of Intellia, said in a press release.
The commission’s decision was based on a positive opinion from the European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products. In October the EMA granted NTLA-2002 PRIME designation, which is meant to accelerate the development of therapies that have the potential to fill an unmet medical need.
Intellia’s experimental therapy also has received innovation passport designation in the U.K., and orphan drug and regenerative medicine advanced therapy designations in the U.S. — all meant to expedite its clinical development and regulatory review.
In HAE, swelling attacks are driven by excessive levels of bradykinin, a signaling molecule that’s produced by an enzyme known as kallikrein.
NTLA-2002 is designed to prevent the production of kallikrein, thereby lowering bradykinin levels and ultimately preventing swelling attacks. The gene-editing therapy specifically works by disrupting the KLKB1 gene, which provides instructions for making a precursor of the kallikrein enzyme.
Intellia is conducting a Phase 1/2 clinical trial (NCT05120830) to test NTLA-2002 in adults with HAE. Interim results from the study’s Phase 1 portion showed the average rate of swelling attacks decreased by 95% after a single dose of the therapy, with nine of the 10 patients being free from attacks for up to nearly one year after treatment.
Phase 2 portion of trial NTLA-2002 is ongoing
The trial’s Phase 2 portion, testing two doses of NTLA-2002 against a placebo, is ongoing. Dosing began earlier this year, and as of late September the trial was still recruiting participants at sites in Australia, New Zealand, France, Germany, the U.K., and the Netherlands. U.S. sites also are expected to open.
“We are on track to complete enrollment of the Phase 2 portion of the study in the coming weeks, which will bring us one step closer to our goal of delivering a potentially life-changing treatment for people who suffer from this serious and debilitating disease,” Leonard said.
Intellia also has announced plans for a Phase 3 trial, expected to start in 2024, that could support approvals of NTLA-2002, should it produce positive results.