日本批准贝罗司他用于遗传性血管性水肿的预防性治疗

(Berotralstat, trade name Orladeyo) is the first oral non-steroidal plasma kallikrein (KLKB1) inhibitor developed by Biocryst. Its IC value for KLKB1 is 5.56 mol·L-1. It was approved by the FDA on December 3, 2020. This product is available in capsule form and is indicated for the prevention of hereditary angioedema (HAE) attacks in adults and pediatric patients 12 years of age and older.

HAE, also known as plasma protein C1 inhibitor deficiency, is characterized by reduced levels or functional defects of C1 inhibitor. It is a rare autosomal dominant genetic disease. HAE can cause pain, weakness, and swelling in the abdomen, throat, face, limbs, and other parts of the body, with clinical manifestations of cutaneous and submucosal edema.

In the phase III APeX-2 trial, beroxostat significantly reduced HAE attacks at week 24 of treatment, and this reduction was sustained through week 48. Among HAE patients who completed 48 weeks of treatment (150 mg), the HAE attack rate was an average of 2.9 attacks per month at baseline and decreased to an average of 1.0 attacks per month after 48 weeks of treatment. In the long-term open-label APeX-S trial, HAE patients who completed 48 weeks of treatment (150 mg) had an average of 0.8 exacerbations per month.

Berostat was safe and well tolerated in 2 trials. The most common adverse reactions reported by patients receiving oral beroxostat compared with placebo were gastrointestinal reactions. These reactions usually occur early after initiation of beroxostat treatment, become less frequent over time, and usually resolve spontaneously.

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