Kallikrein (KLK) is a main rate-limiting enzyme of the kinin system. Specifically, Kallikrein cleaves the substrate peptide at the carbon end. It also releases active kinin, which can regulate cardiovascular system and kidney function. Plasma kallikrein is a kind of kallikrein, participates in the process of coagulation and fibrinolysis. Not only that, plasma Kallikrein acts on high molecular weight kininogen to release bradykinin, regulates vascular tone, inflammation, and fibrinolysis processes. Interestingly, plasma kallikrein plays a significant role in hereditary angioedema (HAE). Therefore, plasma kallikrein has gradually become a research hotspot.

Berotralstat (BCX7353) is a highly specific and orally active plasma kallikrein Inhibitor. It is worthy of note that Berotralstat is the first targeted oral therapy to prevent the onset of HAE. No doubt, the approval of this drug will bring significant progress in the treatment of HAE patients. Specifically, Berotralstat will help reduce the pain of treatment on patients. What exciting is Berotralstat is in the late stage of clinical development for patients to prevent and treat HAE. The combination of efficacy, safety, and tolerability with convenient oral will make berotralstat an important addition to the HAE therapeutic armamentarium. Eventually, a key goal in treating HAE is to allow patients to live a normal life. Providing them with an effective, oral, targeted prophylactic medication is a major step toward that goal.

In conclusion, Berotralstat is a highly specific and orally active plasma kallikrein inhibitor. Berotralstat could provide an effective study for HAE.


[1]. Zuraw B, et al. J Allergy Clin Immunol.2021 Jul;148(1):164-172.e9.