Does Gene Therapy Face Uphill Battle? Gene Therapies for Hemophilia Treatment Will Face Strong Market Headwinds, Research Suggests

Solid market position of Hemlibra and strong prescriber interest in RNAi and TFPI treatment options, combined with patient concerns regarding gene therapies, are likely to create obstacles to market entry.

The latest results from APLUSA’s annual syndicated market surveillance tracker in hemophilia A and B, HEMOPHILIAsyndiTrack™, published in June, show that while unaided awareness of gene therapy for the treatment of both hemophilia A and B is high among physicians – 55% vs. 32% for antithrombin RNAi (RNAi) therapy and 30% for anti-tissue factor pathway inhibitor (TFPI) therapy – the unaided awareness of the specific gene therapies is very low (<5%), while at the same time, 29% of physicians mention Fitusiran (RNAi therapy) and 21% mention Concizumab (TFPI). Despite the generally high awareness of gene therapies, physicians are nearly equally likely to consider prescribing gene therapy within the first year after launch (56%) as they are for RNAi (51%) and TFPI (49%) therapies. Further, the prescribing decision is highly likely to be patient-directed, with physicians basing treatment on the patient’s satisfaction with current therapy.

An interesting study finds that gene therapy will be up against strong competition from increased use of Hemlibra, and similar products coming to market. Visit here for more information.

Source: The Communiqué – August 2022, LA Kelley Communications, Inc.