Drug updated on 3/28/2024
| Dosage Form | Capsule (oral: 110 mg, 150 mg) |
| Drug Class | Plasma kallikrein inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients.
Summary
- Berotralstat (Orladeyo) is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients.
- Two systematic reviews/meta-analyses were reviewed, providing insights into the effectiveness and safety profile of berotralstat compared with other treatments for HAE.
- A network meta-analysis comparing lanadelumab and berotralstat found that lanadelumab 300 mg administered every 2 or 4 weeks was associated with significantly higher effectiveness versus berotralstat 150 mg once daily or 110 mg once daily in reducing HAE attack rate per month and achieving ≥90% reduction in monthly HAE attacks.
- The second review included a total of fifteen studies involving people with Type I and II HAE, investigating several drugs including avoralstat, subcutaneous C1-INH, plasma-derived C1-INH, nanofiltered C1-INH, recombinant human C1-INH, danazol as well as berotralstat; it concluded that all these drugs except avoralstat reduced the number of HAE attacks compared to placebo.
- This same review also reported on quality-of-life outcomes indicating that treatment with Orladeyo improved patient's quality-of-life scores when compared to placebo; additionally it decreased disability related to disease symptoms without increasing risk for adverse events including serious ones.
- Despite its demonstrated efficacy in preventing recurrent episodes of hereditary angioedema among both adult and pediatric populations according to two systematic reviews/meta-analyses documents reviewed - there are still limitations due to lack of direct comparisons between different medications used for this condition which could provide more definitive conclusions about relative efficacy profiles among them especially considering small sample sizes involved within each study conducted.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Orladeyo (berotralstat) Prescribing Information. | 2022 | BioCryst Pharmaceuticals, Inc., Durham, NC |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Clinical and pharmacoeconomic combined report. | 2023 | CADTH |
| Network meta-analysis for indirect comparison of lanadelumab and berotralstat for the treatment of hereditary angioedema. | 2023 | Journal of Comparative Effectiveness Research |
| Interventions for the long-term prevention of hereditary angioedema attacks. | 2022 | The Cochrane database of systematic reviews |
| Assessment report: Orladeyo. | 2021 | EMA |