贝罗司他胶囊可预防12岁以上成人和儿科患者遗传性血管性水肿

Prevents hereditary angioedema in adults and pediatric patients over 12 years of age

HAE is a rare autosomal dominant disease, clinically manifested by recurrent skin and mucosal edema, which can lead to death from suffocation when edema occurs in the respiratory tract. The incidence rate of HAE is only one in 50,000. There are about 10,000 HAE patients in the United States, and about 7,500 patients have been diagnosed and received treatment. The diagnosis rate in China is estimated to be less than 5%, and it generally takes about 13 years for patients to be diagnosed from onset to diagnosis. HAE is caused by a defect or dysfunction of C1 inhibitor, a protein that regulates activation of the classical complement pathway. Lack of or dysfunction of C1 inhibitors can cause elevated bradykinin levels because C1 inhibitors also inhibit activated kallikrein, an enzyme necessary for the metabolism of bradykinin in the kinin system pathway. In December 2020, the FDA approved once-daily oral berosostat capsules for the prevention of hereditary angioedema (HAE) attacks in adults and children over 12 years of age.

Berosostat capsules can prevent HAE attacks by reducing plasma kallikrein activity. In the pivotal Phase 3 clinical trial APeX-2, beroxostat capsules significantly reduced attacks at week 24 and continued through week 48. Patients who completed 48 weeks of treatment (150 mg) experienced a reduction in the number of HAE episodes from an average of 2.9 per month at baseline to 1.0 per month after 48 weeks of treatment.

In the long-term open-label trial APeX-S, patients who completed 48 weeks of treatment (150 mg) had an average number of exacerbations per month of 0.8.

Berosostat capsules demonstrated good safety and tolerability in both trials. The most commonly reported adverse reactions in patients receiving berosostat capsules compared with placebo were gastrointestinal reactions. These reactions usually occur early after starting treatment with beroxostat capsules, become less frequent over time, and usually resolve spontaneously.

HAE treatment can add burden to patients' lives; as a once-daily oral medication, beroxostat capsules can significantly reduce attacks and reduce the burden associated with injections and infusions.

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician.

Recommended related hot articles