Sebetralstat recommended for EU approval for treating HAE attacks
Oral therapy already approved as Ekterly in US for on-demand use
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended that sebetralstat be approved as an on-demand treatment for acute attacks in people with hereditary angioedema (HAE), ages 12 and older.
“This positive CHMP opinion is an important step forward for people living with HAE in Europe, where there remains a high need for effective, easy-to-administer on-demand treatments,” Ben Palleiko, CEO of Kalvista Pharmaceuticals, sebetralstat’s developer, said in a company press release.
The CHMP’s recommendation follows the therapy’s approval earlier this month for the same indication in the U.K and the U.S., where it’s sold under the name Ekterly.
The final decision on whether or not the therapy will be approved in the European Union will be made by the European Commission, and is expected by early October. If approved, sebetralstat will be the first and only oral on-demand HAE therapy in Europe, Kalvista noted.
“Coming just weeks after regulatory approvals in the US and UK, this milestone highlights the potential of sebetralstat to transform how HAE is managed globally,” Palleiko said. “Sebetralstat is expected to become the first and only oral on-demand treatment for HAE available in Europe, bringing forward a new treatment approach that enables adherence to guidelines and empowers people to treat attacks quickly, wherever they occur,” Palleiko said.
Developer says sebetralstat poses less treatment burden for patients
Sebetralstat was designed to mitigate the severity and duration of HAE swelling attacks. It works by inhibiting the activity of kallikrein, an enzyme that plays a key role in the production of bradykinin, the signaling molecule that drives swelling in HAE. By blocking kallikrein, sebetralstat is expected to lower bradykinin levels and bring swelling under control.
According to Kalvista, sebetralstat may ease patients’ treatment burden compared with other on-demand HAE therapies that require injections. It also could minimize treatment delays, the company stated.
The CHMP’s positive opinion is based on data from the global Phase 3 KONFIDENT trial (NCT05259917), which enrolled 136 HAE patients at 66 clinical sites across 20 countries. The results showed that treatment with sebetralstat led to significantly faster symptom relief, reductions in attack severity, and faster symptom resolution relative to a placebo.
The positive CHMP opinion for sebetralstat is an essential move toward a novel, long-awaited oral on-demand option that could help patients treat attacks early and independently, as advocated by guidelines, with the goal of improving outcomes and normalizing lives.
Further analyses from the ongoing KONFIDENT-S (NCT05505916) extension study indicated that sebetralstat rapidly eased swelling attacks, including those rated as severe or very severe and those that could affect the airways.
“Access to on-demand treatments that offer easy self-administration and rapid symptom relief is critical for patients living with HAE,” said Emel Aygören-Pürsün, MD, assistant professor at University Hospital Frankfurt, one of the four trial sites in Germany.
“The positive CHMP opinion for sebetralstat is an essential move toward a novel, long-awaited oral on-demand option that could help patients treat attacks early and independently, as advocated by guidelines, with the goal of improving outcomes and normalizing lives,” Aygören-Pürsün said.
Sebetralstat remains under regulatory review in Japan and other regions.
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