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A review of berotralstat for the treatment of hereditary angioedema
Henriette Farkas1, Zsuzsanna Balla1,2
1Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary.
Insights
Berotralstat, an oral kallikrein inhibitor, offers a new prophylactic treatment for hereditary angioedema (HAE) due to C1-inhibitor deficiency. Clinical trials demonstrate its safety and efficacy in preventing angioedema attacks, improving patients' quality of life.
Area of Science:
- Pharmacology
- Immunology
- Genetics
Background:
- Hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE) significantly impacts patients' quality of life due to unpredictable, potentially life-threatening attacks.
- Effective long-term management of C1-INH-HAE focuses on preventing angioedema attacks to enable patients to lead normal lives.
Approach:
- This review synthesizes data from pivotal clinical trials (APeX-1, ZENITH-1, APeX-2, APeX-S, APeX-J) evaluating the oral kallikrein inhibitor berotralstat.
- Evidence on the efficacy and safety of berotralstat as a targeted oral prophylactic therapy for C1-INH-HAE is reviewed.
Key Points:
- Berotralstat is the first targeted oral prophylactic medication for C1-INH-HAE, approved in 2021.
- Clinical trial data indicate a favorable safety profile and significant efficacy in reducing angioedema attack frequency.
- Berotralstat represents a significant advancement in the long-term management of hereditary angioedema.
Conclusions:
- Berotralstat provides a novel and effective oral prophylactic treatment option for patients with C1-INH-HAE.
- The drug's availability marks a milestone in improving the management and quality of life for individuals with this rare genetic disorder.
Introduction:
Hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE) imposes a significant disease burden on patients and their families. Unpredictable episodes of angioedema, which can lead to life-threatening conditions, have a significant impact on the quality of life of the patient. The fundamental aim of the treatment of C1-INH-HAE is to ensure that patients can lead a normal life. The most effective way to do this is to prevent the onset of angioedema attacks.
Areas Covered:
This review gives a brief overview of the safety and efficacy of the oral kallikrein inhibitor berotralstat in C1-INH-HAE disease. It provides a comprehensive synopsis of the results of the first clinical trials with a targeted oral kallikrein inhibitor (APeX-1 [NCT02870972]; ZENITH-1 [NCT03240133]; APeX-2 [NCT03485911]; APeX-S [NCT03472040]; APeX-J [NCT03873116]), reviewing evidence on the efficacy and safety of the drug, and placing berotralstat on the spectrum of long-term prophylactic therapeutic options.
Expert Opinion:
The availability of the first targeted oral prophylactic drug, the kallikrein inhibitor berotralstat, in 2021, is a milestone in the treatment of patients with hereditary angioedema.
