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Orladeyo (Berotralstat): A Novel Oral Therapy for the Prevention of Hereditary Angioedema
- Source :
- The Annals of pharmacotherapy. 56(4)
- Publication Year :
- 2021
-
Abstract
- Objective The purpose of this article is to review the available trials that led to the Food and Drug Administration (FDA) approval of berotralstat, an oral kallikrein inhibitor, for the prevention of hereditary angioedema (HAE) attacks. Data Sources PubMed and ClincalTrials.gov were searched using key term berotralstat to identify phase III clinical trials related to the FDA approval of berotralstat from April 2018 to May 2021. Study Selection and Data Extraction Trials selected were those that influenced the FDA approval of berotralstat or provided novel information regarding the safety and efficacy of this therapy in the treatment of HAE. Data Synthesis Both APeX-2 and ApeX-J found clinically significant benefit with berotralstat 150 mg daily for reduction in HAE attacks when compared with placebo (1.31 vs 2.35, P < 0.001, and 1.11 vs 2.18, P < 0.001, attacks in the APeX-2 and APeX-J trials, respectively). APeX-2 also showed a statistically significant benefit for berotralstat 110 mg daily (1.65 vs 2.35 attacks [1.65 attacks, P = 0.024]). Relevance to Patient Care and Clinical Practice An advantage berotralstat has over the other approved therapies is that it is administered orally, which may garner patient preference because of ease of administration. Berotralstat has also shown a potential benefit in reducing the need for standard-of-care treatment for HAE attacks, which has not been studied with alternative agents. Conclusions Berotralstat 150 mg daily has been proven safe and effective in clinical studies and appears to be a viable oral alternative to parenteral medications currently used in HAE prophylaxis.
- Subjects :
- 0301 basic medicine
medicine.medical_specialty
Angioedema
business.industry
Angioedemas, Hereditary
Administration, Oral
Kallikrein inhibitor
medicine.disease
Dermatology
Food and drug administration
03 medical and health sciences
030104 developmental biology
0302 clinical medicine
030228 respiratory system
Pharmaceutical Preparations
Hereditary angioedema
Medicine
Humans
Pyrazoles
Pharmacology (medical)
medicine.symptom
business
Oral therapy
Complement C1 Inhibitor Protein
Subjects
Details
- ISSN :
- 15426270
- Volume :
- 56
- Issue :
- 4
- Database :
- OpenAIRE
- Journal :
- The Annals of pharmacotherapy
- Accession number :
- edsair.doi.dedup.....01027a9d031fa47cc98d622c8d239eee