FDA Approves KalVista’s Treatment for Hereditary Angioedema

Rare Daily Staff

The U.S. Food and Drug Administration approved KalVista Pharmaceuticals’ Ekterly as the first and only oral treatment for acute attacks of hereditary angioedema in adult and pediatric patients aged 12 years and older.

Hereditary angioedema (HAE) is a rare genetic disease resulting from a deficiency or dysfunction of the C1 esterase inhibitor protein, leading to uncontrolled activation of the kallikrein-kinin system, which affects a key mediator of swelling. People living with HAE experience painful and debilitating attacks of tissue swelling in various parts of the body, which can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling, shorten the time to attack resolution, and consider treatment for all attacks, regardless of anatomic location or severity.

Ekterly is a novel plasma kallikrein inhibitor. Ongoing studies are exploring its use in children aged two to eleven, and multiple regulatory applications are under review in key global markets.

The efficacy and safety of Ekterly were established by results from KalVista’s phase 3 KONFIDENT clinical trial, the largest clinical trial program ever conducted in HAE. Data from KONFIDENT were published in the New England Journal of Medicine in May 2024, showing that Ekterly achieved significantly faster symptom relief, greater reduction in attack severity, and faster attack resolution than placebo, and was well-tolerated with a safety profile similar to placebo.

The trial randomized 136 HAE patients from 66 clinical sites across 20 countries. These results were further supported by the real-world KONFIDENT-S open-label extension trial, which as of September 2024, showed that Ekterly enabled patients to treat attacks in a median of 10 minutes following onset. The most recent data from KONFIDENT-S show that the onset of symptom relief occurred at a median of 1.3 hours among attacks involving the larynx, the abdomen, and for breakthrough attacks among patients receiving long-term prophylaxis.

The safety profile of Ekterly 600 mg in KONFIDENT-S, in more than 1,700 attacks was consistent with that observed in KONFIDENT.

KalVista will launch Ekterly in the United States immediately, and physicians can begin writing prescriptions today. KalVista has established a comprehensive patient support program that offers personalized services and resources for eligible individuals. This includes assistance with navigating insurance coverage, access support, and ongoing help throughout the treatment journey.

“The FDA approval of Ekterly is a defining moment for people living with HAE,” said Ben Palleiko, CEO of KalVista. “Ekterly enables people to treat attacks the moment symptoms begin, wherever they are.”

Photo: Ben Palleiko, CEO of KalVista