Sebetralstat moves toward early access treatment for HAE in UK

Therapy would be oral alternative to injections for HAE attacks

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has awarded promising innovative medicine designation to sebetralstat, an experimental oral therapy KalVista Pharmaceuticals is developing as an on-demand treatment to resolve swelling attacks in hereditary angioedema (HAE).

The designation is the first step in the Early Access to Medicines Scheme (EAMS), a program that allows patients in the U.K. to access therapies before they’re formally approved. Ben Palleiko, CEO of KalVista, likened the program to the U.S. Food and Drug Administration’s expanded access program allowing patients with serious or immediately life-threatening conditions to take treatments that are not yet approved.

“We are proud to have sebetralstat designated as a promising innovative medicine by the MHRA,” Palleiko said in a company press release, adding the designation “shows that the MHRA believes that we have a promising candidate for the EAMS to treat people living with HAE.”

The move also gives KalVista opportunities for in-depth communication with the U.K.’s health technology agencies and National Health Service.

HAE is characterized by recurrent swelling attacks driven by the overproduction of a signaling molecule called bradykinin. Sebetralstat is designed to reduce bradykinin levels by blocking the activity of kallikrein, an enzyme that controls the production of bradykinin and is overactive in HAE. Sebetralstat aims to reduce swelling during HAE attacks by lowering bradykinin levels.

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Approved, on-demand treatments for HAE attacks given by injection

Approved, on-demand treatments for HAE attacks are all given by injection. KalVista is developing sebetralstat as an oral alternative.

KalVista in February announced positive top-line results from the Phase 3 KONFIDENT trial (NCT05259917), which tested sebetralstat in more than 130 people with HAE type 1 or 2 who were 12 and older.

Most participants given on-demand treatment with sebetralstat saw their symptoms ease in less than two hours, while symptoms for most of those given a placebo didn’t begin to subside for more than six hours. A single dose of sebetralstat was found to be sufficient to ease or resolve the vast majority of swelling attacks.

An open-label extension study, KONFIDENT-S (NCT05505916), is collecting data on the use of on-demand sebetralstat over two years. The trial is open to people with HAE who are 12 and older and have had at least two swelling attacks in the three months before entering the trial. Participants may be on regular prophylactic (preventive) treatment as long as they have been on a stable treatment regimen for at least three months, or at least six months in the case of danazol.

KONFIDENT-S is recruiting new participants at sites worldwide. U.K. sites are in Birmingham, Cambridge, Cardiff, Frimley, Leeds, and London.