On February 3, 2014 the FDA announced their decision to grant breakthrough therapy status to the formidable anemia medication Eltrombopag. The Eltrombopag breakthrough therapy status is based on successful data from a Phase 2 study of Eltrombopag in patients with severe aplastic anemia. The aforementioned study participants were ideal for treatment with Eltrombopag as they had displayed a lack of response to previous immunosuppressive therapy and developed critical cytopenia. While severe aplastic anemia is extremely rare, the use of the nonpeptide thrombopoietin receptor agonist Eltrombopag offers new hope for patients with this uncommon condition. With no other currently approved and available therapies for patients with severe aplastic anemia, the advent of Eltrombopag can aid a large percentage of these patients in life extension. The drug will be marketed under the brand name Promacta/Revolade. A sizable percentage of patients with severe aplastic anemia, about 40%, who do not respond to immunosuppressive therapy will not survive due to excessive bleeding or infection. The use of Eltrombopag gives these hard to treat patients a new chance at recovery.
The Eltrombopag breakthrough therapy status given by the FDA was heavily influenced by the results from a Phase 2 study. This open-label study, which was conducted by the National Institutes of Health involved 43 patients who had demonstrated unresectable severe aplastic anemia, despite extensive prior immunosuppressive therapy. 25 patients enrolled in the Phase 2 study received at least 50 milligrams of Eltrombopag once daily, with dosages based on individual patient needs. The maximum daily dose any of the participants administered with Eltrombopag received was 150 milligrams. Patients in this Phase 2 study were dosed for approximately 12 weeks duration, with endpoints being significant clinical changes in either transfusion dependence or blood count reports. An encouraging 44% of the patients (25 total participants) who were dosed with Eltrombopag had a positive response to their specific dose of this drug, and displayed nominal adverse effects. Researchers concluded that a multilineage clinical response was adequately met in almost half of the patients who received Eltrombopag, thus alleviating their apparent cases of refractory severe aplastic anemia.
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