FDA New Drug Application Accepted for Sebetralstat to Treat Hereditary Angioedema

© KalVista Pharmaceuticals

KalVista Pharmaceuticals Inc. announced today that the FDA has accepted a New Drug Application (NDA) for oral plasma kallikrein inhibitor sebetralstat to treat hereditary angioedema (HAE) attacks in adult and pediatric patients ages 12 and older.The NDA was granted using data from the KONFIDENT phase 3 clinical trial and ongoing KONFIDENT-S open label extension trial. A Prescription Drug User Fee Act (PDUFA) goal date has been set for June 17, 2025.

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 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.

According to the clinical trials, both the 300 mg and 600 mg doses of sebetralstat relieved symptoms significantly faster than placebo. Specifically, results from the KONFIDENT-S trial showed that sebetralstat treats attacks with a median time from attack onset to treatment of 9 minutes. The FDA will not hold an advisory committee meeting to discuss the application.

“We understand that people living with HAE and their families carry a tremendous burden every day as they don’t know when the next attack may occur or if the attack could cause life-threatening consequences,” Ben Palleiko, CEO at KalVista said in a news release. “Given that it could be the first, oral on-demand treatment for HAE, we continue to receive strong support and hear a sense of urgency among healthcare providers, advocates, patients and their families for sebetralstat.”

KalVista’s KONFIDENT-KID clinical trial began in June of this year. It will study the safety and efficacy of sebetralstat in children ages 2 to 11 years old.

In addition, the European Medicines Agency (EMA) validated the submission of the Marketing Authorization Application (MAA) for sebetralstat. KalVista plans to file for approval in the UK and Japan later this year.