Ekterly OK’d as on-demand HAE swelling attack treatment in UK

Therapy led to faster symptom relief, reduced severity in KONFIDENT study

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Ekterly (sebetralstat) has been approved in the U.K. for treating swelling attacks on demand in people with hereditary angioedema (HAE), ages 12 and older, according to its developer, Kalvista Pharmaceuticals.

This makes the therapy the first and only oral on-demand HAE therapy approved in the U.K. It recently earned a similar approval from the U.S. Food and Drug Administration (FDA).

“The approval of Ekterly in the United Kingdom builds on our recent FDA approval and advances our global commitment to bring this innovative treatment option to people living with HAE,” Ben Palleiko, CEO of Kalvista, said in a company press release. “This approval holds particular significance for KalVista as Ekterly was discovered in our Salisbury, U.K. labs, making it even more exciting that now we will be able to offer this therapy to people living with HAE in the U.K.” The decision was made by the Medicines and Healthcare products Regulatory Agency (MHRA).

Ekterly was granted a positive scientific opinion in March for an early access to medicines scheme (EAMS) from the MHRA for HAE attacks, which let healthcare providers prescribe it ahead of its regulatory approval to patients with a clear unmet clinical need. It will remain available under EAMS until the National Institute for Health and Care Excellence (NICE) decides if the therapy will be covered under the U.K.’s public healthcare system. That decision is due in the first half of 2026.

The therapy is also registered with MHRA as an orphan drug, meaning it addresses an unmet need for a rare disease, defined in the U.K. as a condition affecting no more than 5 in every 10,000 people. The designation gives the therapy up to 10 years of market exclusivity.

Ekterly remains under regulatory review in Japan, the European Union, and other markets. Palleiko said Kalvista expects its Ekterly rollout “to be the fastest global launch of an HAE therapy ever.”

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How does Ekterly work in HAE?

Ekterly reduces the severity and duration of HAE’s swelling attacks by blocking the activity of kallikrein, an enzyme involved in producing bradykinin, the molecule that drives HAE attacks. Inhibiting it should lower bradykinin levels and control swelling.

The therapy’s developer believes Ekterly may ease the treatment burden relative to other on-demand HAE therapies, which are given via injections, and also lead to shorter treatment delays.

Ekterly’s approvals are supported by data from the Phase 3 KONFIDENT trial (NCT05259917), where it led to significantly faster symptom relief, reductions in attack severity, and attack resolution over a placebo. Additional analyses from the KONFIDENT-S (NCT05505916) extension study also showed it quickly eased swelling attacks, including severe ones and those that could affect the airways.

“The … approval of Ekterly brings us one step closer to having an oral on-demand treatment available to patients in the U.K. for the first time,” said Sinisa Savic, PhD, a physician and professor at St James’s University Hospital Leeds and a sebetralstat trial investigator. “I look forward to seeing the impact this new treatment option will have on the HAE community in the U.K.”

An ongoing Phase 3 trial called KONFIDENT-KID (NCT06467084) is evaluating the therapy in children with HAE, ages 2-11. The results of the trial, which should finish in 2027, could support its expansion to younger children.