Hemophilia | Niche and Rare Pharmacor | G7 | 2015

Publish date: December 2015

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Hemophilia is a monogenic bleeding disorder occurring primarily in males. In hemophilia A and hemophilia B, factor VIII or factor IX (respectively) is either absent or deficient in function. Several factor replacement serums are currently approved to treat hemophilia A and B, but a comparatively large unmet need remains for hemophilia A patients because of disappointing factor half-life extensions, compounded by the higher diagnosed prevalence of hemophilia A vs B. Launch of the new longer-acting factor concentrates and agents with novel mechanisms of action, for both type A and B, offer a chance to achieve improvements in clinical outcomes and address compliance issues. Using primary research conducted with expert and European hemophilia specialists, this report provides a comprehensive analysis of the competitive landscape and market opportunity for hemophilia. It includes a comprehensive analysis of patient populations, current therapies and medical practices, unmet needs, and emerging therapies.

Questions Answered in This Report:

  • Congenital hemophilia is a monogenic bleeding disorder, inherited in an X-linked recessive pattern and therefore occurring primarily in males. What is the size of the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom) hemophilia A and B patient population, and how will it change over the next ten years?

  • Treatment of hemophilia may involve replacing the missing or deficient factor, using factor replacement therapy. What are the limitations of existing hemophilia treatments? Are physicians favoring plasma-derived or recombinant products, and how will this practice change through 2025? How will physicians’ practice change with the advent of the longer-acting factor concentrates in hemophilia A and B, and which patient groups will they target for switching? Will there still be a place in therapy for the standard half-life products? How do physicians perceive gene therapy for hemophilia A vs B?

  • The largest unmet need lies in hemophilia A. What do physicians think of the new pegylated molecules and will this view impact patient share? Given the disappointing half-life extensions of FVIII molecules, is there a place for pharmacokinetic tailoring in hemophilia A? How might the hemophilia A treatment algorithm change through 2025?

  • Inhibitor patients remain one of the major challenges in hemophilia. What are the limitations of current therapies, and how might emerging therapies address physician concerns? What do physicians think of the Chugai/Roche agent ACE 910, and how do they anticipate they will use it in their practice?

Scope:

Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: Nine country-specific interviews with thought leaders and physicians specializing in hemophilia.

Epidemiology: Diagnosed prevalent cases of hemophilia A and B, split by severity, gender, and age. Diagnosed prevalence of inhibitors, split by type of hemophilia (A vs B), severity, and titer.

Emerging therapies: Phase II: 3 drugs; Phase III: 6 drugs; preregistration: 3 drugs.

Author(s): Kerri Brown, M.Pharm.
Sarah Anderson, M.Sc.