“Breakthrough Therapy” designation for GlaxoSmithKline’s Promacta/Revolade for SAA

The US Food and Drug Administration has granted “Breakthrough Therapy” designation for UK pharma giant GlaxoSmithKline’s (LSE: GSK) Promacta/Revolade (eltrombopag) for the treatment of cytopenias in patients with severe aplastic anemia (SAA) who have had insufficient response to immunosuppressive therapy (IST).

Eltrombopag is not approved or licensed anywhere in the world for use in this treatment setting, although it has been cleared for marketing in other indications, such as idiopathic thrombocytopenic purpura, since 2008. Breakthrough Therapy Designation is the newest of the FDA’s programs aimed at accelerating the development and review times of drugs for serious or life-threatening conditions.

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 initial immunosuppressive therapy. Of those patients unresponsive to initial IST, around 40% die from infection or bleeding within five years of their diagnosis.