Sebetralstat up for approval in Japan as on-demand HAE treatment
Japanese regulatory agency awards oral therapy orphan drug status
Kalvista Pharmaceuticals has submitted an application asking Japan’s Ministry of Health, Labour and Welfare (MHLW) to approve its oral therapy sebetralstat as an on-demand treatment to manage swelling attacks in people with hereditary angioedema (HAE), ages 12 and older.
“The submission of our [new drug application] for sebetralstat in Japan represents another key step toward our efforts to make this important new treatment available to as many people living with HAE as possible,” Ben Palleiko, CEO of KalVista, said in a company press release.
Kalvista also announced that the MHLW has granted sebetralstat orphan drug status. This designation is awarded to therapies intended to treat fewer than 50,000 people in Japan, with the goal of giving extra incentives to companies that are working to develop treatments for serious diseases for which there is a high unmet medical need. Among the incentives conferred by this designation are tax credits, subsidies, and faster regulatory review.
“The orphan drug designation not only acknowledges the critical need for new, effective treatments for HAE in Japan but underscores the potential of sebetralstat to provide meaningful relief for people who have faced ongoing challenges with existing options,” Palleiko said.
HAE treatment sebetralstat sped time to symptom relief in trial
HAE is a genetic disorder marked by bouts of swelling that are driven by the excessive production of a signaling molecule called bradykinin. Several on-demand treatments are available to manage HAE swelling attacks, but all of them are administered via injection. If approved, sebetralstat would become the first oral on-demand therapy for HAE. The oral medicine works to block the activity of kallikrein, an enzyme involved in the production of bradykinin.
Besides Japan, Kalvista has submitted applications seeking the approval of sebetralstat in the U.S. and the European Union, as well as in Australia, Singapore, Switzerland, and the U.K.
Kalvista’s applications seeking the therapy’s approval are based on data from the Phase 3 KONFIDENT trial (NCT05259917), completed in 2023, and its extension study KONFIDENT-S (NCT05505916). These studies tested sebetralstat in adults and children, ages 12 and older, with HAE types 1 or 2.
Results from the studies showed that sebetralstat accelerated the time to symptom relief compared with a placebo, with patients typically starting to experience symptom relief within two hours of taking sebetralstat.
A separate Phase 3 study called KONFIDENT-KID (NCT06467084) is testing sebetralstat in children with HAE types 1 or 2, ages 2 to 11. That study is still recruiting participants at sites in the U.S., Canada, France, Germany, Israel, and Italy.
“We are proud to be at the forefront of advancing care for the HAE community,” Palleiko said.
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