FDA approves first-of-a-kind oral therapy for rare disease
The US drug agency’s authorisation provides hereditary angioedema (HAE) patients with the first new on-demand treatment in over ten years.
The US Food and Drug Administration (FDA) has approved the first oral on-demand therapy for acute attacks of hereditary angioedema (HAE) in individuals aged 12 years and older.
This rare genetic disease leads to tissue swelling attacks in the body that can be life-threatening. It 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) “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.”
“Until now, on-demand treatment for [hereditary angioedema] relied on injectable subcutaneous or intravenous administration, often resulting in delayed intervention”
The Phase III KONFIDENT clinical trial, part of the largest clinical trial programme conducted 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 results for the small molecule therapy are further supported by data from the extension KONFIDENT-S clinical trial. As of September 2024, attacks could be treated with Ekterly in a median of 10 minutes following onset.
