The US Food and Drug Administration (FDA) has approved the first on-demand oral therapy for the treatment of acute attacks of hereditary angioedema (HAE) in people aged 12 years and older.
Treating hereditary angioedema is crucial, which is a rare genetic disease, responsible for tissue swelling attacks within the body that may prove to be life-threatening. The disease is caused by a deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system, KalVista Pharmaceuticals shared.
The company’s novel plasma kallikrein inhibitor Ekterly® (sebetralstat) helps in treating hereditary angioedema “enables people to treat attacks the moment symptoms begin, wherever they are,” commented Ben Palleiko, CEO of KalVista Pharmaceuticals.
Significance of the FDA’s hereditary angioedema treatment approval
“This is an important moment for patients, giving people living with HAE a treatment option that could provide greater independence and control over managing their condition,” explained Dr Marc Riedl, Professor of Medicine and Clinical Director, U.S. Hereditary Angioedema Association Center at the University of California, San Diego, and an investigator for the KONFIDENT Phase III trial.
“Until now, on-demand treatment relied on injectable subcutaneous or intravenous administration, often resulting in delayed intervention. Having an oral option empowers patients to treat attacks early, which aligns with treatment guidelines and advances our goal as physicians to reduce the overall burden of disease.”
The Phase III KONFIDENT clinical trial, which is part of the largest clinical trial program ever being conducted out in HAE, found that Ekterly “achieved significantly faster symptom relief, reduction in attack severity and attack resolution than placebo, and was well-tolerated with a safety profile similar to placebo”, KalVista Pharmaceuticals shared.
These outcomes for the small molecule therapy are also being supported by evidence from the extension of the KONFIDENT-S clinical trial. Through September 2024, attacks were being treated with Ekterly in a median of 10 minutes after onset.
