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Long‐term safety and effectiveness of berotralstat for hereditary angioedema: The open‐label APeX‐S study
- Source :
- Clinical and Translational Allergy, Vol 11, Iss 4, Pp n/a-n/a (2021), Clinical and Translational Allergy, Farkas, H, Stobiecki, M, Peter, J, Kinaciyan, T, Maurer, M, Aygören-Pürsün, E, Kiani-Alikhan, S, Wu, A, Reshef, A, Bygum, A, Fain, O, Hagin, D, Huissoon, A, Jeseňák, M, Lindsay, K, Panovska, V G, Steiner, U C, Zubrinich, C, Best, J M, Cornpropst, M, Dix, D, Dobo, S M, Iocca, H A, Desai, B, Murray, S C, Nagy, E & Sheridan, W P 2021, ' Long-term safety and effectiveness of berotralstat for hereditary angioedema : The open-label APeX-S study ', Clinical and Translational Allergy, vol. 11, no. 4, e12035 . https://doi.org/10.1002/clt2.12035, Clinical and Translational Allergy, BioMed Central, 2021, 11 (4), ⟨10.1002/clt2.12035⟩
- Publication Year :
- 2021
- Publisher :
- Wiley, 2021.
-
Abstract
- Background: Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein recently approved for prevention of angioedema attacks in adults and adolescents with hereditary angioedema (HAE). The objective of this report is to summarize results from an interim analysis of an ongoing long-term safety study of berotralstat in patients with HAE. Methods: APeX-S is an ongoing, phase 2, open-label study conducted in 22 countries (ClinicalTrials.gov, NCT03472040). Eligible patients with a clinical diagnosis of HAE due to C1 inhibitor deficiency (HAE-C1-INH) were centrally allocated to receive berotralstat 150 or 110 mg once daily. The primary objective was to determine long-term safety and the secondary objective was to evaluate effectiveness. Results: Enrolled patients (N = 227) received berotralstat 150 mg (n = 127) or 110 mg (n = 100) once daily. The median (range) duration of exposure was 342 (11–540) and 307 (14–429) days for the 150-mg and 110-mg groups, respectively. Treatment-emergent adverse events (TEAEs) occurred in 91% (n = 206) of patients. The most common TEAEs across treatment groups were upper respiratory tract infection (n = 91, 40%), abdominal pain (n = 57, 25%), headache (n = 40, 18%), and diarrhea (n = 31, 14%) and were mostly mild to moderate. Fifty percent (n = 113) of patients had at least one drug-related adverse event (AE; 150 mg, n = 57 [45%]; 110 mg, n = 56 [56%]), and discontinuations due to AEs occurred in 19 (8%) patients (150 mg, n = 13 [10%]; 110 mg, n = 6 [6%]). Three (1.3%) patients experienced a drug-related serious TEAE. Among patients who received berotralstat through 48 weeks (150 mg, n = 73; 110 mg, n = 30), median HAE attack rates were low in month 1 (150 mg, 1.0 attacks/month; 110 mg, 0.5 attacks/month) and remained low through 12 months (0 attacks/month in both dose groups). Mean HAE attack rates followed a similar trend, and no evidence for patient tolerance to berotralstat emerged. In both dose groups, angioedema quality of life scores showed clinically meaningful changes from baseline. Conclusions: In this analysis, both berotralstat doses, 150 and 110 mg once daily, were generally well tolerated. Effectiveness results support the durability and robustness of berotralstat as prophylactic therapy in patients with HAE. Trial registration: The study is registered with ClinicalTrials.gov (NCT03472040).
- Subjects :
- safety
Pulmonary and Respiratory Medicine
medicine.medical_specialty
Abdominal pain
Immunology
610 Medicine & health
030207 dermatology & venereal diseases
03 medical and health sciences
0302 clinical medicine
Quality of life
Internal medicine
medicine
Immunology and Allergy
Adverse effect
long-term
2403 Immunology
Angioedema
business.industry
Research
[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology
RC581-607
medicine.disease
Interim analysis
berotralstat
hereditary angioedema
3. Good health
Diarrhea
Upper respiratory tract infection
030228 respiratory system
2740 Pulmonary and Respiratory Medicine
Hereditary angioedema
10033 Clinic for Immunology
2723 Immunology and Allergy
long‐term
prophylaxis
Immunologic diseases. Allergy
medicine.symptom
business
Subjects
Details
- ISSN :
- 20457022
- Volume :
- 11
- Database :
- OpenAIRE
- Journal :
- Clinical and Translational Allergy
- Accession number :
- edsair.doi.dedup.....e81f42d82c32ab05143c0be9d9ea1878