CSL Behring’s gene therapy for hemophilia B, HemGenix, was evaluated in the HOPE-B Phase III trial versus a basket of conventional FIX replacement products where it was found that the gene therapy reduced the annual bleed rate (ABR) versus conventional therapy. However, this trial was not powered to detect differences in the performance of HemGenix versus individual FIX replacement products such as Alprolix or IDELVION.
To answer this question, CSL Behring, clinical colleagues, and EVERSANA authors including Ashley Bonner, Kirk Szafranski, Di Wang, and Sarah Walsh collaborated on an indirect treatment comparison (ITC) to evaluate the HemGenix efficacy from the HOPE-B versus efficacy from FIX replacement therapy trials (such as B-LONG for the product Alprolix). The results of this ITC confirmed that HemGenix reduced bleeding versus all individual comparators as measured by ABR and therefore offers a more efficacious treatment compared with currently available therapies for Hemophilia B.
To read the full journal article, please visit https://pubmed.ncbi.nlm.nih.gov/37902714/.
To read the American Journal of Managed Care’s review of this study, please visit: https://www.ajmc.com/view/etranacogene-dezaparvovec-gene-therapy-outperforms-fix-products-in-hemophilia-b.
Author
Kirk Szafranski is a Director, HEOR in the Value & Evidence Division at EVERSANA. Kirk Szafranski holds an M.Sc. in Laboratory Medicine and Pathobiology from the University of Toronto and has extensive experience with…