Marrowforums - Promacta Receives Breakthrough Therapy Status in the United States

Pharmaceutical company GlaxoSmithKline announced on February 3, 2014 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy status to their severe aplastic anemia (SAA) drug eltrombopag, which GlaxoSmithKline markets under the trade name Promacta in the U.S. and as Revolade in other countries.

Promacta/Revolade is already approved for treatment of thrombocytopenia (low platelets) in patients with chronic idiopathic thrombocytopenic purpura (ITP) in both the U.S. and Europe. In studies, Promacta has also been found to help aplastic anemia patients who have had immunosuppressive treatment with horse or rabbit ATG and still have very low platelets.

The FDA grants Breakthrough Therapy designation to treatments for life-threatening diseases (like SAA) when the treatment appears to provide "substantial improvement over existing therapies". Promacta will now receive expedited development and review support from the FDA, an important step toward FDA approval.

The FDA's action was based in part on A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Aplastic Anemia Patients With Immunosuppressive-Therapy Refractory Thrombocytopenia, an ongoing clinical trial at the National Institutes of Health.

Although patients can receive drugs that are not FDA-approved for their disease through off-label usage or in clinical trials, FDA approval leads to wider U.S. availability and better insurance coverage, as well as increased confidence in a drug's safety and efficacy.