Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn’t just a mere hypothetical but a significant struggle. Hereditary angioedema results from a genetic defect on chromosome 11 that causes episodes of edema (swelling caused by fluid accumulation) throughout the body. People may experience extremities swollen to twice their normal size, intestinal tract swelling leading to severe nausea and abdominal distention, or life-threatening throat swelling that may cause respiratory distress. As a result, people with HAE experience not just physical discomfort, but quality-of-life disruptions.

In recent years, medical advancements have paved the way for innovative treatments aimed at alleviating the burden of HAE. However, these treatments often come with potential side effects or barriers to care such as requiring multiple injections or only reducing, not preventing, HAE attacks. Continuing research into treatment development is necessary to improve care and quality-of-life for those affected.

What This Phase 3 Trial Could Mean for HAE

Sebetralstat, developed by KalVista Pharmaceuticals (“KalVista”), is one of these promising developments. KalVista describes sebetralstat as:

an investigational novel, orally-administered therapy for the on-demand treatment of HAE. Sebetralstat works by targeting the kallikrein-kinin system (KKS) cascade, selectively inhibiting plasma kallikrein and its uncontrolled activity that drive HAE attacks.

According to Ben Fidler of Biopharma Dive, sebetralstat performed well in a Phase 3 study evaluating the therapy for hereditary angioedema. Fidler notes that KalVista released summary findings from the study which suggest that:

• Sebetralstat effectively reduced the severity of HAE attacks quicker than a placebo did. The median time to response was 1.6 hours for low-dose sebetralstat and 1.8 hours for high-dose sebetralstat.
• Researchers found sebetralstat to be safe and well-tolerated, with no serious reactions related to treatment.
• 40% of those treated with sebetralstat needed a second dose, with 10% of those treated requiring rescue medication within 12 hours of the sebetralstat treatment.

KalVista plans to release more comprehensive data on the study findings later this year. Additionally, if all goes well, the company plans to file for FDA approval of this treatment.