Berotralstat reduces overall disease burden in hereditary angioedema: Study

USA: In a new study conducted by Emel Aygoren-Pursun and team, it was found that berotralstat, regardless of baseline attack rate, is an effective oral preventive therapy option that can minimize disease burden against hereditary angioedema (HAE). The findings of this study were presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) 2022 Annual Meeting.

It is well established that lowering attack rates reduces the overall illness burden of people with HAE. Prophylactic HAE therapy aims to lessen the disease burden by lowering attack rates. Berotralstat is a preventive once-daily (QD) therapy for HAE. The long-term effectiveness of berotralstat 150mg in patients who finished 96 weeks of therapy in the APeX-2 trial (NCT03485911), stratified by baseline attack rate, has been published in this study.

For 24 weeks, patients were randomly assigned to berotralstat (110mg or 150mg) or placebo QD. Patients assigned to berotralstat stayed on the same dose at Week 24, while placebo patients were rerandomized to berotralstat for a further 24 weeks; following Week 48, all patients remained on berotralstat 150mg. Twenty-one individuals received berotralstat 150mg for a total of 96 weeks. This study divided patients into tertiles based on their baseline attack rate: Group 1: 2 attacks per month; Group 2: 2 to 3 attacks per month; Group 3: 3 attacks per month.

The results of this study stated as follow:

1. The mean (SEM) monthly assault rate in Group 1 (n57) decreased from 1.2(0.1) at baseline to 0.3(0.2) at Week 24, 0.1(0.1) at Week 48, and 0 at Week 96.

2. In Group 2 (n57), the mean monthly assault rate fell from 2.6(0.2) at baseline to 1.1(0.5) at Week 24, 0.1(0.1) at Week 48, and 0.3(0.2) at Week 96.

3. The mean attack rate in Group 3 (n57) decreased from 4.6(0.6) at baseline to 1.7(0.8) at Week 24, 1.6(0.6) at Week 48, and 0.7(0.4) at Week 96.

4. Over 70% of patients in each tertile saw a >70% relative reduction in attack rate.

In conclusion, regardless of the baseline attack rate, berotralstat is a primary oral preventive therapy option that can minimize disease burden by reducing HAE-related deficits.

Reference:

Aygoren-Pursun E, McNeil D, Collis P, et al. Oral Berotralstat Treatment for 96 Weeks Consistently Reduces Hereditary Angioedema (HAE) Attack Rates Regardless of Baseline Attack Rate. Presented at: American Academy of Allergy, Asthma & Immunology (AAAAI) 2022 Annual Meeting; February 25–28, 2022; Phoenix, AZ. Abstract 491.