is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII.
People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life threatening. There are an estimated 16,000 – 20,000 people in the United States with hemophilia A (and 142,000 world wide).
Several orphan approved drugs are available to treat bleeding episodes and 3 orphan drugs - Advate
- are approved for prophylactic use in patients.
Acquired hemophilia A
is a rare autoimmune bleeding disorder caused by the development of autoantibodies, in adult life, that attacks Factor VIII. Although both disorders involve deficiency of the same clotting factor, the bleeding pattern is different.
Acquired hemophilia is usually spontaneous and an underlying cause is not clear. It has been associated with hematological cancers, allergic drug reaction, and in some women as a postpartum complication. Unlike classic hemophilia, bleeding into the joints is rare. Instead, muscles, soft tissue, and mucous membranes are more susceptible.
Acquired hemophilia affects 2 per 1,000,000 Americans per year. The incidence increases with age and most cases affect elderly individuals.
Two orphan drugs are approved for acquired hemophilia A - Obizur
[antihemophilic factor (recombinant)] is approved for the treatment of bleeding episodes in adults with acquired hemophilia A and Novoseven
(coagulation factor VIIa) is approved for prevention of bleeding in surgical interventions or invasive procedures in patients with acquired hemophilia.