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 who are unresponsive to initial immunosuppressive therapy (IST).
In February 2014, Ligand announced that GSK had gained Breakthrough Therapy designation for Promacta in Severe Aplastic Anemia. Also in February 2014, GSK announced the submission of a supplemental New Drug Application (sNDA) to the FDA for Promacta for the treatment of cytopenias (a reduction in blood cells) in patients with SAA who have had an insufficient response to IST.
The sNDA is based on the results from an open-label, Phase II National Institute of Health (NIH) study (09-H-0154) of eltrombopag in 43 heavily pre-treated SAA patients with an insufficient response to IST.