GlaxoSmithKline has announced that FDA has approved a supplemental new drug application (sNDA) for the once-daily use of Promacta (eltrombopag) in patients with severe aplastic anaemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST).
SAA is a blood disorder where the bone marrow fails to make enough red blood cells, white blood cells and platelets. Eltrombopag, an oral thrombopoietin (TPO) receptor agonist, works by helping to induce proliferation and differentiation of bone marrow stem cells to increase production of blood cells.
"FDA approval of Promacta addresses a significant treatment need for this very rare but serious blood disorder in those who have failed current treatment options," said Dr Paolo Paoletti, president of oncology, GSK. "Through collaboration with the National Institutes of Health, whose studies demonstrate the potential for Promacta to achieve a haematologic 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."
Promacta gained breakthrough therapy designation status from FDA in January 2014 and priority review in April 2014. Today's approval by the FDA is based on results from an investigator-sponsored Phase II study (09-H-0154) conducted by the National Heart, Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH). The study demonstrated a haematologic response in SAA patients treated with eltrombopag who had an insufficient response to IST:1