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Hemophilia Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2018


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Hemophilia Pipeline Drugs


Hemophilia Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2018



Hemophilia is a rare blood disorder, in which blood doesn’t clot normally due to lack of sufficient clotting factors. Hemophilia A and B both are inherited diseases. Hemophilia A, B caused due to deficiency of clotting factor VIII,IX respectively. Hemophilia causes by genetic mutation. The mutations that involve genes that code the clotting factors.

Hereditary hemophilia can be easily diagnosed during the pregnancy if the fetus is suffering from hemophilia. In absence of hereditary hemophilia, disease may be due to the spontaneous mutations in gene. Hemophilia in both children and adults, blood test to determine the clotting factor may be preferred. Treatment depends on the type of hemophilia.



By type, hemophilia pipeline drugs are segmented into

  • Hemophilia A
  • Hemophilia B

By trial phase, hemophilia pipeline drugs are segmented into

  • Preclinical Studies
  • Phase-I
  • Phase-II
  • Phase-III
  • Phase-IV

By mechanism, hemophilia pipeline drugs are segmented into

  • Inhibitors
  • Factor Concentrates
  • Gene Therapy
  • Non-Factor Concentrates

By company, hemophilia pipeline drugs assessment is segmented into

  • Pfizer Inc. (U.S.)
  • Hoffmann-La Roche AG (Switzerland)
  • Novo Nordisk A/S (Denmark)
  • Shire Plc. (Republic of Ireland)
  • Alnylam Pharmaceuticals (U.S.)
  • OPKO Health (U.S.)


Space Analysis:

Major companies are focused on the development of novel therapies for the treatment of hemophilia. Many pipeline drugs under development in various phases of clinical trials. Most of the companies are focused on the development of factor concentrates to replace the deficient clotting factors with recombination technology or other biologic technologies. Furthermore, pipeline has Gene therapy products, non-factor concentrates and inhibitors with promising ongoing trails.

  • In November 2017, Roche (Genentech)announced positive results for its PhaseIII trial (HAVEN III) in patients withhemophilia A without inhibitors. Theonce‐weekly Hemlibra therapy reportedsuperiority in reducing treated bleeds tofactor VIII prophylaxis in an intra‐patientcomparison.
  • In April 2017, Catalyst Biosciences announced it had received approval from the KoreanMinistry of Food and safety to beginhuman clinical trials for its newinvestigational drug CB2679dISU304 for treatment of hemophilia B.


Report Description:

Hemophilia Pipeline Drugs Assessment report studies the various therapeutics under clinical development for hemophiliatreatment along with targets for various drug candidate. The report provides plethora of information pertaining to trail phases, companies involved in the hemophilia pipeline drugsdevelopment. This report studies the dynamics of the Hemophilia Pipeline Drugs i.e. drivers, challenges and opportunities which are significantly impacting the product development. The report provides various information pertaining the clinical trials such as designation, grants, patents, and technology among others. Moreover, the report on hemophilia pipeline drugs assessment comprehensively presents the geographic location, trial status information along with key players involved in the therapeutics development.


Key Features of the Report:

  • Provides the information related to universities and research institutes working in the therapeutics development
  • Report comprehensively covers the all active and discontinued studies
  • Studies the entire pipeline with special emphasis on companies actively involved in the therapeutics development
  • Presents the prominent targets for drug development in each stage of clinical trial
  • Provides the in-depth analysis onthe each drug candidates in the clinical trial phases
  • Pfizer Inc. (U.S.)
  • Hoffmann-La Roche AG (Switzerland)
  • Novo Nordisk A/S (Denmark)
  • Shire Plc. (Republic of Ireland)
  • Alnylam Pharmaceuticals (U.S.)
  • OPKO Health (U.S.)

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