Hemophilia | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2016

DRG anticipates the next ten years to be an exciting time in the hemophilia space, as the number of treatment options grow for patients with, and without, inhibitors. The hemophilia A pipeline is diverse, both in terms of technology type and administration route, and we are witnessing a shift in R&D emphasis; there is growing interest in non-factor treatment approaches to restore coagulation. Examples include the launch of Roche/Chugai’s bispecific antibody Hemlibra, and the development of gene therapies, such as Biomarin’s BMN 270, and the anti-TPFI drug class, with agents like Novo Nordisk’s concizumab. Despite novel non-factor hemophilia agents forecast to launch, DRG expects the high demand for factor concentrates to persist. We anticipate the factor concentrate market will remain highly competitive, and pipeline agents are looking to further evolve this treatment approach.


  • How will physicians treating hemophilia differentiate between the various emerging therapies, and which will gain the greatest market and patient share in an increasingly stringent regulatory environment?
  • What clinical role will Hemlibra, fitusiran, and gene therapy play in the evolving hemophilia A treatment landscape?
  • How will the management of patients with inhibitors change with the arrival of new treatment options? How will Hemlibra’s head start on its competitors play out over the forecast period?
  • What can entrenched hemophilia players do to further protect their factor-based franchises from non-factor agent competition?
  • What will be the market impact of gene therapies in hemophilia ?


Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.


United States, EU5, Japan


20 country-specific interviews with thought-leading hematologists, specializing in hemophilia. Supported by survey data collected for this and other DRG research


Alnylam, Aptevo, Bayer, Biomarin, Bioverativ, Chugai, CSL Behring, Dimension, Novo Nordisk, Octapharma, OPKO, Pfizer, Roche, Sangamo, Sanofi, Shire, Spark, uniQure


Advate, Adynovate/Adynovi, Afstyla, Alprolix, AMT-061, Benefix, BMN 270, concizumab, DTX 201, Eloctate/Elocta, FEIBA, fitusiran, Helixate, Hemlibra, Idelvion, Ixinity, Kogenate, Kovaltry/Iblias, N8-GP, NovoEight, NovoSeven, Nuwiq, OPK88005, PF-06741086, Rebinyn/Refixia, ReFacto/Xyntha, Rixubis, SB-525, SB-FIX, SPK-8011, SPK-9001

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