贝罗司他美国获批预防遗传性血管性水肿(HAE)发作

Berotralstat is an oral capsule approved to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older. Most HAEs are caused by a deficiency (type 1 HAE) or dysfunction (type 2 HAE) of a protein called C1 esterase inhibitor. It is a plasma kallikrein inhibitor that binds to plasma kallikrein and inhibits its proteolytic activity. Recently, berosostat was approved in the United States to prevent hereditary angioedema (HAE) attacks. Let’s take a look at it below.

Hereditary angioedema (HAE) is a rare autosomal dominant genetic disease, clinically characterized by recurrent and unpredictable cutaneous and submucosal edema. Edema can occur at any location, and the most fatal is upper respiratory tract mucosal edema, which can lead to respiratory tract edema. Blockage, causing suffocation or even life-threatening; secondly, when edema involves the gastrointestinal mucosa and other abdominal organs, it can manifest as severe abdominal pain, accompanied by nausea and vomiting, which is difficult to distinguish from surgical acute abdomen and may lead to unnecessary abdominal surgery. Therefore, prevention and treatment of this disease is urgent.

Berotralstat capsules (150 mg once daily) significantly reduced the number of HAE episodes over 24 weeks in patients in the pivotal Phase 3 APeX-2 trial, and this continued through week 48. After completing the 48-week trial, essentially all patients experienced a significant reduction in the frequency of HAE attacks, from an average of 2.9 attacks/month at baseline to an average of 1 attack/month. It can be seen from this that the therapeutic effect of Berostat capsule is very excellent.

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