Hereditary Angioedema Oral Treatment

My Take: Oral delivery adds to treatment options, though price may become a limiting factor.

Name: Ekterly (sebetralstat)

Class: Oral plasma kallikrein inhibitor

Usefulness: Treatment of hereditary angioedema (HAE) attacks in adult and adolescent patients aged 12 years and older, marking the first oral option available for acute management of this rare and potentially life-threatening swelling disorder.

Method of Action: Inhibits plasma kallikrein, an enzyme that plays a central role in the production of bradykinin, a mediator of the painful and swelling symptoms in HAE.

Potential Market: HAE affects around 8,000 people in the U.S., with annual sales estimated to be $250 million.

Competing Agents: Existing therapies for acute HAE include injectable options such as:

• Firazyr (icatibant, a bradykinin receptor antagonist)

• Intravenous C1-inhibitor concentrates (e.g., Berinert, Cinryze)

Cost to Consumer: About $11,000 per dose, slightly higher than Firazyr.

Unusual Fact: While hereditary angioedema is a genetic condition, it may present in individuals without any known family history.