The following article is a part of conference coverage from the American Academy of Allergy, Asthma & Immunology Virtual Annual Meeting, being held virtually from February 26 to March 1, 2021. The team at Pulmonology Advisor will be reporting on the latest news and research conducted by leading experts in the field. Check back for more from the AAAAI 2021 Virtual Annual Meeting.


Berotralstat may reduce the use of on-demand medications in patients with hereditary angioedema (HAE) who were previously treated with prophylactic therapies, according to research presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Virtual Annual Meeting, held February 26 to March 1, 2021.

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The purpose of HAE prophylaxis is to minimize the number of HAE attacks and the associated disease burden, including treatment with on-demand medications. Berotralstat is an oral once-daily selective plasma kallikrein inhibitor that has been shown to reduce attack frequency in a phase 3 study ( Identifier: NCT03485911).

In part 1 of this double-blind, placebo-controlled study, researchers evaluated the reduction in on-demand medication use by randomly assigning individuals to receive berotralstat 110 mg (n=41): berotralstat 150 mg (n=40): placebo (n=40) daily for 24 weeks. For this post hoc analysis, patients were grouped according to which prior prophylaxis they had received: prior C1 esterase inhibitor (C1-INH), prior androgen, or no prior prophylactic medication (however, prior C1-INH and prior androgen categories were not mutually exclusive).

In patients with prior C1-INH prophylaxis (berotralstat 150 mg, n=21; placebo, n=16), the rate of use of on-demand treatment was significantly reduced compared with placebo (-59.2%, P =.002). Similar reductions were found in patients with prior androgen use (berotralstat 150 mg, n=22; placebo, n=25; -51.8%; P =.004) and in patients without prior prophylaxis (berotralstat 150 mg, n=10; placebo, n=10; -71.2%; P =.019). The rate reduction corresponded to approximately 2.2 fewer doses of on-demand medication per month compared with placebo in patients with prior C1-INH use, 1.6 for those with prior androgen use, and 1.4 for those with no prior prophylaxis.

“Prophylactic treatment with oral berotralstat 150 mg resulted in significant reductions in on-demand medication compared to placebo irrespective of prior prophylactic treatment,” the study authors concluded.

Disclosure: Several study authors declared affiliations with the pharmaceutical industry. Please see the original reference for a full list of authors’ disclosures.


Gower R, Busse P, Best J, Murray S, Iocca H, Kinaciyan T. Berotralstat reduces use of on-demand medication in hereditary angioedema (HAE) patients previously treated with prophylactic therapies. Presented at: the American Academy of Allergy, Asthma & Immunology (AAAAI) Virtual Annual Meeting; February 26-March 1, 2021. Abstract 465.

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