Is Berotralstat an Effective Oral Prophylactic for Hereditary Angioedema?

woman with swollen eye
woman with swollen eye
APeX -2 trial findings presented at AAAAI 2022 examine the efficacy of berotralstat, in varying doses, as an oral prophylactic for reducing HAE attacks.

The following article is a part of conference coverage from the American Academy of Allergy, Asthma & Immunology Annual Meeting, being held in Phoenix, Arizona, from February 25 to 28, 2022. Click here to read more of MPR‘s conference coverage.

A recent study of the drug berotralstat shows that it is efficacious as an oral prophylactic treatment in reducing hereditary angioedema (HAE) attack rates, irrespective of baseline attack rate. Study findings are being presented at the American Association of Allergy, Asthma & Immunology (AAAAI) 2022 Annual Meeting, held in Phoenix, Arizona, from February 25 to 28.

Reducing attack rates is known to lower the overall disease burden of patients with HAE. Researchers tested the long-term efficacy of berotralstat, a once-a-day (QD) prophylactic treatment for the disorder, by administering 150 mg to patients who finished 96 weeks of treatment in the APeX -2 trial ( Identifier: NCT03485911), ranked by baseline attack rate.

The investigators randomized patients to berotralstat (110mg or 150mg) or placebo QD for 24 weeks. At week 24, patients receiving berotralstat stayed on the same dose, and placebo patients were re­randomized to berotralstat for 24 weeks longer. After week 48, all patients remained on berotralstat 150mg. In all, 21 patients finished a total of 96 weeks of the drug regimen. This study evaluated patients by tertiles of baseline attack rate in 3 groups: (1) less than 2 attacks per month, (2) 2 to less than 3 attacks per month, and (3) 3 attacks per month.

In group 1 patients (n=7), the mean monthly attack rate fell from 1.2 (standard error [SE], 0.1) at baseline to 0.3 (SE, 0.2) at week 24, 0.1 (SE, 0.1) at week 48, and 0 at week 96. Group 2 (n =7) patients experienced a mean monthly attack rate decline from baseline 2.6 (SE, 0.2) to 1.1 (SE, 0.5) at week 24, 0.1 (SE, 0.1) at week 48, and 0.3 (SE, 0.2)  at week 96. In group 3 (n=7), the mean attack rate dropped from a baseline 4.6 (SE, 0.6) to 1.7 (SE, 0.8) at week 24, 1.6 (SE, 0.6) at week 48, and 0.7 (SE, 0.4) at week 96.

More than 70% of patients in each tertile had an approximate 70% relative  decline in attack rate. As the researchers concluded, “Regardless of baseline attack rate, berotralstat is an effective oral prophylactic treatment option that can reduce disease burden.” They added that by reducing “the impairments associated with HAE,” berotralstat provides patients with a less burdensome treatment option.

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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.                                       

This article originally appeared on Pulmonology Advisor