News Feature | November 17, 2014

GSK Files EU Marketing Authorization Application For Eltrombopag In Anemia

By Estel Grace Masangkay

GlaxoSmithKline announced that it has submitted a marketing authorization application to the European Medicines Agency (EMA) for eltrombopag to include an additional indication for the treatment of severe aplastic anemia (SAA).

Eltrombopag is an oral thrombopoietin (TPO) receptor agonist indicated for the treatment of thrombocytopenia associated with chronic immune thrombocytopenic purpura (ITP), as well as thrombocytopenia associated with chronic hepatitis C infection. The drug works by helping induce proliferation and differentiation of bone marrow stem cells, in turn bolstering production of blood cells. The new EU filing seeks approval for adult patients with severe aplastic anaemia (SAA) who are unresponsive to immunosuppressive therapy (IST).

The drug is marketed as Revolade in the EU and is known as Promacta in the U.S., where it recently earned approval from the U.S. Food and Drug Administration (FDA) as a once-daily treatment for patients with SAA who have had an insufficient response to IST. Eltrombopag was approved by the FDA under Breakthrough Therapy Designation and Priority Review status.

The company’s filing with the EMA is supported by results of a pivotal open-label Phase 2 study as well as two supporting Phase 2 studies assessing eltrombopag in patients with SAA. The studies were conducted in collaboration with the National Institutes of Health (NIH).

Severe aplastic anemia is a very rare, serious blood disorder wherein the bone marrow is unable to produce sufficient red blood cells, white blood cells, and platelets. Although the exact root cause of SAA is not fully understood, most cases of SAA are considered to be triggered by an autoimmune response that attacks blood-forming stem cells found in the bone marrow. Patients with SAA are at heightened risk of fatal infections or bleeding. There are no established standard of care for patients with SAA who are unable to respond to IST or who are not qualified for hematopoietic stem cell transplantation.