Clarivate expects the next 10 years to be transitional for patients with hemophilia A (with and without inhibitors) owing to the entry of several novel therapeutic options into the market. 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 the approach to disease management of many patients. The emphasis in R&D has shifted away from factor-VIII-replacement-based approaches to new mechanisms aimed at restoring coagulation, including gene therapies. These new therapies include Sanofi’s fitusiran and BIVV001, Novo Nordisk’s concizumab, Pfizer’s marstacimab, and BioMarin’s Roctavian. Despite the various novel nonfactor hemophilia agents forecast to launch, we expect the demand for factor-based concentrates to persist.
QUESTIONS ANSWERED
How has Hemlibra changed the treatment landscape for hemophilia A for patients with or without inhibitors?
How will hematologists differentiate between the various emerging therapies?
What clinical roles will BIVV001, fitusiran, concizumab, marstacimab, mim8, and gene therapy play in the evolving hemophilia A treatment landscape?
How will the management of patients with or without inhibitors change when new treatment options become available?
How will the factor VIII concentrate market evolve? How will nonfactor treatment options affect the multitude of brands vying for patient share in this space?
CONTENT HIGHLIGHTS
Markets covered: United States, France, Germany, Italy, Spain, United Kingdom, Japan.
Primary research: 25 country-specific interviews with thought-leading hematologists. Supported by survey data collected for this and other Clarivate research.
Epidemiology: Diagnosed prevalence of hemophilia A +/- inhibitors by country; population segmentation by disease severity and inhibitor titer.
Emerging therapies: Coverage of select emerging therapies from preclinical to preregistration stages.
Market forecast features: 10-year, annualized, drug-level sales and patient share of key hemophilia A (and inhibitor) therapies through 2031, segmented by brands and epidemiological subpopulations.
PRODUCT DESCRIPTION
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.
SOLUTION ENHANCEMENT
Disease Landscape & Forecast will feature continual updates in 2022 to provide timely insights and analyses as meaningful indication-specific news and events unfold.
Additionally, Disease Landscape & Forecast introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.
Expectations for launch and sales opportunity of Roctavian (BMN 270; valoctocogene roxaparvovec) in hemophilia A
Key results of select clinical trials investigating giroctocogene fitelparvovec for the treatment of hemophilia A
Key ongoing clinical trials of giroctocogene fitelparvovec in the treatment of hemophilia A
Analysis of the clinical development program for giroctocogene fitelparvovec
Expectations for launch and sales opportunity of giroctocogene fitelparvovec in hemophilia A
Key results of select clinical trials investigating SPK-8011 for the treatment of hemophilia A
Key ongoing clinical trials of SPK-8011 in the treatment of hemophilia A
Analysis of the clinical development program for SPK-8011
Expectations for launch and sales opportunity of SPK-8011 in hemophilia A
Key results of select clinical trials investigating BAY2599023 for the treatment of hemophilia A
Key ongoing clinical trials of BAY-2599023 (DTX-201) in the treatment of hemophilia A
Analysis of the clinical development program for BAY-2599023 (DTX-201)
Expectations for lunch and sales opportunity of BAY-2599023 (DTX-201) in hemophilia A
Early-phase pipeline analysis
Select compounds in early-phase development for hemophilia A
Early-phase pipeline analysis
Select compounds in early-phase development for hemophilia A
Key discontinuations and failures in hemophilia A
Expert insight: key discontinuations and failures
Access & reimbursement overview
Region-specific reimbursement practices
Key market access considerations in hemophilia A+/- inhibitors: United States
General reimbursement environment: United States
Key market access considerations in hemophilia A+/- inhibitors: EU5
General reimbursement environment: EU5
Key market access considerations in hemophilia A+/- inhibitors: Japan
General reimbursement environment: Japan
Appendix
Abbreviations
Hemophilia bibliography
Yogesh Shelke
Yogesh Shelke, M.B.A. (Pharm.), Manager, Cardiovascular, Metabolic, Renal, and Hematology. Before joining Clarivate, he was instrumental in providing actionable insights to clients from various leading pharmaceutical companies regarding competitive intelligence, market sizing and forecasting, pipeline analysis, situational analysis, unmet need analysis, and market entry strategies in a variety of therapeutic areas. He holds a bachelor’s degree in pharmacy from Pune University and a master’s degree in pharmaceutical marketing from the National Institute of Pharmaceutical Education and Research in Mohali, India.
Shilpa Thakur
Shilpa Thakur, M.P.H., is an epidemiologist at Clarivate. Previously, she monitored HIV sentinel surveillance in Himachal Pradesh. She has also studied the patterns of antimicrobial resistance in India. She received her M.P.H. from the Postgraduate Institute of Medical Education and Research with a specialization in epidemiology and biostatistics.