FDA Approves Ekterly, First Oral Treatment for Hereditary Angioedema

KalVista Pharmaceuticals Inc. announced today that the FDA has approved Ekterly (sebetralstat), a novel plasma kallikrein inhibitor for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients, according to a news release. Ekterly is now the first and only oral, on-demand treatment for HAE patients. Orsini will be the specialty pharmacy partner for Ekterly, a separate news release says. Ekterly is available in the United States immediately, and physicians can start writing prescriptions today.

HAE is a rare genetic disorder caused by a dysfunction in the C1 esterase inhibitor (C1INH) protein, which is part of a group of proteins in the body’s immune system. As a result, periods of severe and sometimes deadly swelling follow. The parts of the body most affected are the limbs, face, intestinal tract and airway. Episodes may be triggered by anxiety, medical procedures or illnesses such as a cold or flu, but often episodes are difficult to predict. Episodes often occur without warning and can travel within the body. Early signs of an attack include extreme fatigue, tingling of the skin and hoarseness of the voice, according to the American Academy of Allergy, Asthma and Immunology.

Ekterly comes in the form of a 300 mg tablet. The recommended dosage is 600 mg split into two doses taken at the earliest sign of an attack, according to the prescribing information. Additional tablets may be taken if symptoms do not improve, with a maximum recommended dosage of 1,200 mg in a 24-hour period.

The efficacy of Ekterly was determined during the phase 3 KONFIDENT clinical trial, the largest HAE clinical trial to date, which included 136 HAE patients from 66 clinical sites across 20 countries. Approximately 43% of patients treated with 300 mg had complete attack resolution within 24 hours, and approximately 50% of patients treated with 600 mg had complete attack resolution within 24 hours. Results were published in the New England Journal of Medicine in May 2024. The most recent data from KONFIDENT-S showed that symptom relief started within approximately 2 hours, whereas placebo took approximately 7 hours, according to the news release.

The safety of Ekterly was determined in 110 patients aged 12 years and older with HAE. Ekterly treated 264 attacks. The most common adverse reaction was headache, occurring in at least 2% of patients.

Ekterly has a wholesale acquisition price of $16,720, according to data presented during an investors call this morning.

Patient support is available through the KalVista Cares program. Eligible commercially insured patients can pay as little as $0 for their prescription, with up to an annual maximum benefit of $40,000. Patients covered by Medicare, Medicaid,
Veterans Affairs or other federal or state health plans are not eligible for this program.

“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, M.D., professor of medicine and clinical director of the U.S. Hereditary Angioedema Association Center at the University of California, San Diego, and an investigator for the KONFIDENT phase 3 trial, said in the news release. “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.”