CAMBRIDGE, Mass.– The U.S. Food and Drug Administration has approved EKTERLY® (sebetralstat), making it the first and only oral on-demand treatment for hereditary angioedema (HAE) in patients 12 and older, KalVista Pharmaceuticals announced Monday.
EKTERLY, a plasma kallikrein inhibitor, is intended to treat acute HAE attacks, which often occur unpredictably and can be life-threatening. The approval is based on data from the KONFIDENT phase 3 trial, the largest study of its kind in HAE, which showed rapid symptom relief, a favorable safety profile, and consistent performance across different types of attacks.
“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. This approval affirms the strength of our science and deep commitment to the HAE community.”
Prior to EKTERLY, on-demand HAE treatments in the U.S. required either intravenous or subcutaneous injection, which KalVista says can delay treatment and complicate management of the disease.
“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,” said Marc A. Riedl, M.D., Professor of Medicine and Clinical Director at the U.S. Hereditary Angioedema Association Center at the University of California, San Diego.
Riedl, an investigator in the KONFIDENT trial, emphasized that oral treatment can reduce the burden of disease by enabling earlier intervention. Data from KONFIDENT, published in The New England Journal of Medicine in May 2024, showed EKTERLY provided significantly faster symptom relief and attack resolution compared to placebo. KalVista reported that the medication was well tolerated, with a safety profile similar to placebo.
Results from the KONFIDENT-S open-label extension trial also support the drug’s performance in real-world conditions. As of September 2024, patients reported treating attacks a median of 10 minutes after onset, with symptom relief beginning in about 1.3 hours for laryngeal, abdominal, and breakthrough attacks.
“As the first orally administered on-demand therapy for HAE attacks, EKTERLY provides patients and physicians with an important and welcome advance in HAE treatment options,” said Anthony J. Castaldo, CEO of the U.S. Hereditary Angioedema Association.
KalVista said EKTERLY will be available immediately, and prescriptions can be written starting today. The company has also launched KalVista Cares™, a patient support program offering assistance with insurance navigation, access coordination, and other personalized services.
