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    Groundbreaking gene therapies in store for hemophilia


    Hemophilia is an inherited bleeding disorder that prevents a person’s blood from clotting properly and is caused by a mutation in the genes. The most common types of hemophilia are hemophilia A (lack of clotting factor VIII) and hemophilia B (lack of clotting factor IX).

    Hemophilia predominantly affects males and according to the CDC, 20,000 Americans have it.


    “Although the population of patients with hemophilia is small, cost of treatment for an individual patient can be extremely high, from tens of thousands of dollars for each monthly prescription to over a million dollars per year,” says Crystal Blankenship, PharmD, senior clinical consultant for Accredo specialty pharmacy’s bleeding disorders therapeutic resource center.

    The CDC estimates that about 15% to 20% of patients with hemophilia develop inhibitors that prevent treatments from working, further complicating therapy.

    Current treatments

    Current treatments include both recombinant and plasma-derived direct factor replacements that require intravenous administration, says Blankenship.

    Market leaders for hemophilia A include:

    • Advate (antihemophilic factor [recombinant])
    • Eloctate (zntihemophilic factor [recombinant], Fc fusion protein)
    • Humate-P (antihemophilic factor/von Willebrand factor complex [human])
    • Adynovate (antihemophilic factor [recombinant], PEGylated)
    • Novoeight (antihemophilic factor [recombinant])
    • Kovaltry (antihemophilic factor [recombinant])

    BeneFix, a recombinant factor IX treatment, is the market leader in the hemophilia B space. Other commonly used factor IX products include:

    • Idelvion (coagulation factor IX [recombinant], albumin fusion protein [rFIX-FP])
    • Alprolix (coagulation factor IX [recombinant], FC fusion protein)
    • Mononine (coagulation factor IX)
    • Ixinity (coagulation factor IX [recombinant])

    Next: New and emerging therapies


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