FDA Approves Sebetralstat for Hereditary Angioedema
The therapy is now the first and only oral on demand treatment option for the condition.
The FDA has approved sebetralstat (Ekterly) for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age or older, according to a release from KalVista Pharmaceuticals.1 The decision makes sebetralstat the first and only oral on demand treatment option for the condition.
FDA Approves Sebetralstat For Hereditary Angioedema / Grandbrothers - stock.adobe.com
The approval was based on data from the phase 3 KONFIDENT trial (NCT05259917), in which the therapy met its primary endpoint of time to beginning of symptom relief patient global impression of change. Sebetralstat also met all key secondary endpoints, including time to first incidence of decrease from baseline patient global impression of severity and time to complete HAE attack resolution.
“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,” Marc A Riedl, MD, an investigator on the trial, said in a release.1 “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.”
KONFIDENT was a randomized, double-blind, placebo-controlled, 3-way crossover phase 3 clinical trial that evaluated the efficacy and safety of sebetralstat for the treatment of hereditary angioedema attacks in adolescent and adult patients. The study cohort included 136 patients with a confirmed HAE type 1 or 2 diagnosis who had at least 2 documented HAE attacks within 3 months prior to screening. Patients received either 300 mg or 600 mg sebetralstat or placebo.
The study found patients who received sebetralstat had a beginning of symptom relief of 1.61 hours for 300 mg and 1.79 hours for 600 mg, compared to 6.72 hours for placebo. The patients who received the therapy also had a significant reduction in attack severity and quicker attack resolution compared to placebo. Additionally, the therapy was seen to be well tolerated with a similar safety profile as placebo.2
KalVista said in the release sebetralstat will be launched in the United States immediately so physicians can quickly begin writing prescriptions. The company also said it established a comprehensive support program that offers personalized services and resources for eligible patients.
“The FDA approval of Ekterly is a defining moment for people living with HAE,” Ben Palleiko, CEO of KalVista, said in a release.1 “Ekterly enables people to treat attacks the moment symptoms begin, wherever they are. This approval affirms the strength of our science and deep commitment to the HAE community…Ekterly has the potential to become the foundational treatment for HAE and our focus now is on delivering it to the people who need it.”
READ MORE: FDA Updates Resource Center
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