Sebetralstat under approval review in EU as on-demand HAE therapy
If approved, treatment would be first oral way of managing swelling attacks
Authorities in the European Union (EU) have agreed to review Kalvista Pharmaceuticals’ application requesting the approval of oral sebetralstat as an on-demand treatment for swelling attacks in people with hereditary angioedema (HAE).
The company’s marketing authorization application (MAA) now will be reviewed by a committee of the European Medicines Agency for all 27 EU members states, as well as for Norway, Iceland, and Liechtenstein.
“The validation of this MAA, which we submitted in July, brings us another step closer to our goal of delivering sebetralstat on a global scale to people living with HAE,” Ben Palleiko, CEO of Kalvista, said in a company press release.
Swelling attack relief quickly followed therapy’s use in clinical trials
“If approved, sebetralstat would be the first oral, on-demand treatment for HAE in Europe,” Palleiko added. “We believe it has the potential to become the foundational treatment for this disease, addressing many underlying unmet needs in the community.”
Kalvista recently submitted an application to U.S.regulators, seeking sebetralstat’s approval as an on-demand treatment for HAE patients, ages 12 and older. The company said it expects to make similar requests to authorities in other countries, including the U.K. and Japan, later this year.
The swelling attacks that characterize HAE are due to mutations that lead to the overproduction of a signaling molecule called bradykinin. Sebetralstat is designed to block the activity of kallikrein, the enzyme that mediates the production of bradykinin. By blocking kallikrein, sebetralstat is expected to lower bradykinin levels and ultimately control swelling.
Other treatments are available for the on-demand management of HAE attacks, but they are all given by injection. Sebetralstat is poised to become the first oral on-demand therapy for managing HAE swelling attacks.
Kalvista’s approval requests were based mainly on data from the Phase 3 KONFIDENT clinical trial (NCT05259917) and the KONFIDENT-S extension study (NCT05505916). These studies tested sebetralstat in HAE patients ages 12 and older, with results showing that most patients started to experience symptom relief in less than two hours after being given the oral therapy.
Kalvista recently launched a separate clinical trial, called KONFIDENT-KID (NCT06467084), into sebetralstat as an on-demand treatment of children with HAE, ages 2-11. That study is actively recruiting patients at six sites in the U.S., and it is open to children with HAE types 1 or 2 who have had at least one swelling attack in the previous year.
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