According to a story from pm360online.com, the U.S. Food and Drug Administration (FDA), has given Priority Review status to eltrombopag (marketed as Promacta) after impressive clinical trial results. Eltrombopag was developed by Novartis and is designed to treat severe aplastic anemia. The review is for the use of eltrombopag in combination with the current standard of treatment for aplastic anemia, immunosuppressive therapy.
Aplastic anemia is a rare disease that inflicts damage to the bone marrow as well as the stem cells that are located there. This affects the ability of the stem cells to create blood cells and causes a deficiency of red blood cells, white blood cells, and platelets. Anemia causes symptoms such as palpitations, pale skin, and malaise (general feeling of discomfort/unwellness). Deficiencies in platelets can increase the risk of bruising and bleeding, and deficiencies in white blood cells can make the patient more vulnerable to infection. Causes are unknown in some cases, but they are mostly environmental; exposure to radiation, certain chemicals, drugs, infections, and immune system disease are all potential causes. The disease is lethal if left untreated, but adequate treatment with medication and stem cell transplant gives a five year survival of 85 percent. To learn more about aplastic anemia, click here.
Eltrombopag has already been approved for patients in the refractory period who have not had an adequate response to immunosuppressive therapy (IST), but this new approval could significantly expand the drug’s indication and make it available to a larger pool of patients. Recent studies indicate that 52 percent of patients who used the treatment alongside immunosuppressive therapy achieved a total response after six months, which is 35 percent greater in comparison to patients who used immunosuppressive therapy alone. The total response rate after six months was also good, at 85 percent.
