KalVista Pharmaceuticals, Inc. has achieved a transformative breakthrough in the treatment of hereditary angioedema (HAE) with the FDA approval of EKTERLY® (sebetralstat). This marks a pivotal moment as EKTERLY becomes the first and only oral on-demand treatment available for acute HAE attacks in adult and pediatric patients aged 12 years and older, revolutionizing a landscape previously dominated by burdensome injectable therapies. HAE is a rare genetic disorder characterized by unpredictable and painful swelling attacks in various body parts, caused by an uncontrolled activation of the kallikrein-kinin system due to a deficiency or dysfunction of the C1 esterase inhibitor protein. EKTERLY directly addresses this by acting as a novel plasma kallikrein inhibitor, a small molecule designed to be rapidly absorbed orally and selectively block the overactive plasma kallikrein enzyme, thereby preventing the excessive production of bradykinin, which is responsible for the debilitating swelling. The drug's efficacy and well-established safety profile were validated in KalVista's extensive Phase 3 KONFIDENT clinical trial, the largest ever conducted in HAE, which demonstrated significantly faster symptom relief and attack resolution across diverse patient populations, regardless of attack severity, location, age, or use of long-term prophylaxis. This data was further supported by the real-world evidence from the KONFIDENT-S open-label extension trial, showing patients could treat attacks within a median of 10 minutes from onset, with symptom relief beginning in a median of 1.3 hours for various attack types.

For KalVista Pharmaceuticals, this approval represents the culmination of deep scientific commitment and perseverance, positioning the company as a leader in rare disease therapeutics. The immediate U.S. launch of EKTERLY is underway, with physicians now able to prescribe this new oral option. KalVista is also prioritizing patient access and support through its comprehensive KalVista Cares™ program, offering personalized services from insurance navigation to ongoing treatment assistance. This strategic move aims to ensure that HAE patients can readily access and benefit from EKTERLY, fostering greater independence and control over their condition by enabling early intervention at the first sign of symptoms. With ongoing studies exploring its use in children aged two to eleven and multiple regulatory applications under review in key global markets, KalVista is strategically expanding EKTERLY's reach, with the ambition for it to become the foundational therapy for HAE management worldwide, fundamentally improving the lives of individuals affected by this challenging disease.