FDA Approves GSK's Promacta Label Expansion In SAA

GlaxoSmithKline reported that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for the additional indication of Promacta (eltrombopag) in patients with severe aplastic anemia (SAA) who have had an inadequate response to immunosuppressive therapy (IST).

Eltrombopag is indicated for the treatment of thrombocytopenia in certain patients with chronic immune (idiopathic) thrombocytopenia (ITP) or chronic hepatitis C. The FDA designated eltrombopag Breakthrough Therapy status in January. GSK and its collaborator Ligand Pharmaceuticals’ application was also accepted for Priority Review in April.

Dr. Paolo Paoletti, President of Oncology at GSK, said, “Through collaboration with the National Institutes of Health, whose studies demonstrate the potential for Promacta to achieve a hematologic response in at least one lineage – red blood cells, platelets, or white blood cells – patients now have a treatment option where one didn’t previously exist.”

Severe aplastic anemia is a rare and life-threatening blood disorder in which the bone marrow’s ability to produce red blood cells, platelets, and white blood cells is diminished. The cause of SAA is yet to be completely understood but is considered to be triggered by an autoimmune response wherein the immune system attacks the blood-forming stem cells in the bone marrow. Patients with SAA are at risk for serious infections or bleeding. Around 300 to 600 new cases of SAA are diagnosed every year in the U.S.

Standard treatments include immunosuppressive therapy (IST) or hematopoietic stem cell transplantation. As of present, SAA patients who had insufficient response to IST or are ineligible for hematopoietic stem cell transplantation have no established standard of care options.

Eltrombopag’s approval was based on the positive results of the single center, single arm, open label Phase II study where eltrombopag achieved a hematologic response in patients treated with the drug. The study was sponsored and conducted by the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH).