Favourable results for Elocta when evaluating treatment options for haemophilia A.- SOBI

The international medical journal Advances in Therapy has recently published data evaluating treatment options for haemophilia A . The journal reported that when using individualised prophylaxis with extended half-life (EHL) products, Elocta (rFVIIIFc) from Swedish Orphan Biovitruum (SOBI) resulted in lower annual bleed rates when compared to Jivi (BAY 94-9027). The report was based on an indirect comparison of pivotal clinical trial data in adults. "We are conscious of the difficulties to conduct clinical head-to-head studies in a rare disease setting such as this," said Jennifer Cain-Birkmose, Global Head of Patient Access and Community Engagement. "Through utilising the validated MAIC research method, this analysis provides important data to further understand treatment options and demonstrates Elocta's value for people living with haemophilia," she said. Results : Mean annualized bleeding rate (ABR) was lower in the rFVIIIFc individualised prophylaxis group versus the BAY 94-9027 pooled prophylaxis population (3.0 vs 4.9). This difference (-1.9; 95% CI -3.5, -0.4) was both clinically relevant and statistically significant (p=0.02).The proportion of patients with zero bleeds was numerically higher for rFVIIIFc (45.5%) than for BAY 94-9027 (38.2%), although the difference was not statistically significant (odds ratio: 1.35; 95% CI 0.77, 2.36). After matching, the effective sample size for A-LONG was 81 people with haemophilia A. rFVIIIFc was approved by the European Commission in November 2015 for patients with haemophilia A of all age groups. BAY 94-9027 was approved three years later for the treatment of haemophilia A in patients older than 12. About the MAIC analysis : Matching-adjusted indirect comparison (MAIC) is a research method adopted by health technology assessment bodies around the world, including the National Institute for Care Excellence (NICE). The analysis was performed to evaluate the relative efficacy of both extended half-life therapies for the prophylactic treatment of haemophilia A. The indirect comparison builds on data from the individualised prophylaxis arm of the A-LONG phase III clinical trial evaluating rFVIIIFc in 117 people with haemophilia A, and from the pooled prophylaxis population of the PROTECT VIII phase II/III study evaluating BAY 94-9027 in 110 people with haemophilia A. To adjust for cross-study differences in baseline characteristics, propensity score weighting was used to balance demographic and clinical characteristics. Outcomes assessed from the A-LONG study and the PROTECT VIII study included ABR and percentage of patients with no bleeds during the study period. As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria. See- Hakimi Z, Santagostino E, Postma MJ, Nazir J." Recombinant FVIIIFc Versus BAY 94-9027 for Treatment of Patients with Haemophilia A: Comparative Efficacy Using a Matching Adjusted Indirect Comparison". Adv Ther. 2020 Dec 30. doi: 10.1007/s12325-020-01599-1. Epub ahead of print. PMID: 33377987..