Drug intelligence / Profile preview

berotralstat

Development stage
Approved
Lead developer
BioCryst Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

Berotralstat is an oral small molecule inhibitor of plasma kallikrein, developed for the prophylaxis (prevention) of attacks in patients with hereditary angioedema (HAE), a rare genetic disorder characterized by recurrent episodes of severe swelling. By binding to and inhibiting plasma kallikrein, berotralstat reduces the production of bradykinin—a potent vasodilator responsible for increased vascular permeability and swelling in HAE. Berotralstat is indicated for use in adults and pediatric patients aged 12 years and older to prevent HAE attacks. It is not used for acute treatment of attacks. The drug was developed by BioCryst Pharmaceuticals and is marketed under the brand name Orladeyo.

Brand names
Orladeyo
Other names
berotralstatBCX7353BCX-7353BCX 7353Orladeyo
02

Targets

KLKB1 (Plasma kallikrein)

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