GSK files for US approval for Promacta for severe aplastic anemia

UK pharma giant GlaxoSmithKline (LSE:GSK) has submitted a supplemental New Drug Application to the US Food and Drug Administration for Promacta (eltrombopag) for the treatment of cytopenias (a reduction in blood cells) in patients with severe aplastic anemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST).

SAA is a rare disorder in which the bone marrow fails to make enough new blood cells. There are no approved therapies available for SAA patients unresponsive to IST. Of those patients unresponsive to initial IST, around 40% die from infection or bleeding within five years of their diagnosis.

A month ago, GSK announced that the FDA granted Breakthrough Therapy designation for Promacta in SAA (The Pharma Letter February 3).The drug, which is partnered with USA-based Ligand Pharmaceuticals (Nasdaq: LGND) and marketed in Europe and other countries as Revolade, in 2013 generated sales of £186 million ($310 million), up 46% year-on-year.