MATCHING-ADJUSTED INDIRECT TREATMENT COMPARISON BETWEEN LANADELUMAB AND BEROTRALSTAT FOR HEREDITARY ANGIOEDEMA PROPHYLAXIS

Abstract

<h3>Introduction</h3> To date, no head-to-head studies compared the efficacy of lanadelumab and berotralstat for the prevention of hereditary angioedema (HAE) attacks. In this analysis, matching-adjusted indirect comparison (MAIC) was used to estimate comparative effectiveness of lanadelumab versus berotralstat over 48 weeks of treatment as there were no common comparators to link a network of evidence. <h3>Methods</h3> The MAIC used the data from the HELP open-label extension (HELP OLE; NCT02741596) and APeX-2 (NCT03485911) studies. To preserve randomization, patients from the HELP OLE Study were re-weighted to match the clinically relevant baseline characteristics from the APeX-2 study. The efficacy outcome of interest was the HAE attack rate per 28 days. <h3>Results</h3> In this analysis, lanadelumab 300 mg every 2 weeks (Q2W) was associated with a statistically significantly 73% lower HAE attack rate per 28 days compared with berotralstat 150 mg QD over 48 weeks of treatment (rate ratio [RR] 0.27; 95% CI 0.24, 0.31; p<0.00001). In a subgroup analysis that only considered patients from the HELP OLE Study who rolled over from lanadelumab 300 mg Q2W arm in the HELP Study, lanadelumab 300 mg Q2W was associated with a statistically significantly 81% lower HAE attack rate per 28 days compared with berotralstat 150 mg QD over 48 weeks of treatment (RR 0.19; 95% CI 0.12, 0.29; p<0.0001). <h3>Conclusion</h3> In this MAIC, lanadelumab 300 mg Q2W was associated with a statistically significantly lower rate of HAE attacks per 28 days versus berotralstat 150 mg QD at 48 weeks of treatment.