Business Expanding KalVista Pharmaceuticals receives Orphan Drug Designation and Submits New Drug Application for Sebetralstat in Japan for Hereditary Angioedema

KalVista Pharmaceuticals Japan K.K. was established in January 2024 as a Japanese subsidiary of KalVista Pharmaceuticals, Inc. in order to prepare for a new drug application and subsequent launch of sales of sebetralstat in Japan.

The NDA submission is supported by previously disclosed results, including data from the KONFIDENT phase 3 clinical trial and ongoing KONFIDENT-S open-label extension trial. In a placebo-controlled, randomized, double-blind, cross-over, phase III study, sebetralstat achieved all primary and main secondary endpoints and demonstrated a favorable safety profile. Sebetralstat is under regulatory review by the U.S. FDA, with a PDUFA goal date of June 17, 2025. In addition, the Company has completed Marketing Authorization Applications for sebetralstat to multiple global regulatory authorities.

On January 21, 2025, KalVista announced that sebetralstat was designated as an orphan drug (*1) by the MHLW, and a new drug application was submitted in January 2025.

If approved, sebetralstat would be the first oral on-demand treatment for HAE in Japan.

About HAE

Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the location affected. All currently approved on-demand treatment options require either intravenous or subcutaneous administration.

To assist with the establishment of the company's Japanese base, JETRO's Invest Japan Business Support Center (IBSC) provided temporary office space and multi-language information on registration, labor and tax, and also provided support services for corporate public relations.