DRG expects the next ten years to be transitional for patients with hemophilia A, with and without inhibitors, as both the number of therapy options and size of the hemophilia market increase. With the emergence of Roche / Chugai’s bispecific antibody Hemlibra and a diverse pipeline, both in terms of technology type and administration route, we forecast a significant change in approach to disease management for many patients. The R&D emphasis has shifted to nonfactor treatment approaches to restore coagulation. Examples include Sanofi’s anti-ATIII siRNA fitusiran, Novo Nordisk’s anti-TFPI MAb concizumab, and BioMarin’s gene therapy BMN 270. Despite novel nonfactor hemophilia agents forecast to launch, we expect the demand for factor-based concentrates to persist.
- How will physicians treating hemophilia A differentiate between the emerging therapies? Which therapies will gain the greatest market share in an increasingly stringent regulatory environment?
- What clinical roles will Roche / Chugai’s Hemlibra, Sanofi’s fitusiran, Novo Nordisk’s concizumab, and gene therapy play in the evolving hemophilia A treatment landscape?
- How will the management of patients with inhibitors change when new treatment options become available? How will Hemlibra’s head start on its novel competitors play out over the forecast period?
- How will the factor VIII concentrate market evolve? How will nonfactor treatment options impact the multitude of brands vying for patient share in this space?
- What do interviewed payers advise developers to do to drive market access of new hemophilia drugs? To what extent will the cost of FVIII gene therapy drive its rationing in terms of a patient’s capacity to derive benefit?
Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.