The MHRA has approved sebetralstat (Ekterly) as the first oral, on-demand treatment for hereditary angioedema (HAE) attacks in patients aged 12 and above.
Sebetralstat allows patients to treat attacks at the earliest sign of symptoms without injections, offering greater convenience and faster intervention.
HAE is a rare genetic disorder that causes unpredictable and painful swelling in areas such as the face, throat and abdomen. Attacks can be life-threatening if they affect the airways.
Sebetralstat works by blocking the release of bradykinin, a substance that causes blood vessels to leak fluid and trigger swelling. The medicine is taken orally in tablet form.
Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, said: “Patient safety is our top priority, which is why we are pleased to approve sebetralstat for the treatment of hereditary angioedema attacks (HAE).”
He added: “HAE attacks can have a sudden and serious impact on people’s lives, often requiring immediate intervention. This approval is the first and only MHRA-approved oral option for treating HAE attacks, which will be valuable for patients who prefer not to use injectable treatments.”
The approval is based on data from the KONFIDENT trial, which involved 110 patients and 264 treated attacks. Results showed that sebetralstat significantly shortened the time to symptom relief and full resolution compared to placebo.
Sebetralstat’s availability marks a major step forward in HAE care, offering patients a fast-acting and user-friendly alternative to injectable therapies. The MHRA will continue to monitor its safety as part of routine post-approval surveillance.
