PROMACTA(R) (eltrombopag) RECEIVES UNANIMOUS RECOMMENDATION BY FDA ADVI...(n affecting less than 200000 individuals...),Health

Eltrombopag is an investigational, once-daily oral treatment developed to induce the production of cells in the bone marrow to increase platelets, which are critical in minimizing the incidence of bleeding in chronic ITP.

Eltrombopag, a novel oral thrombopoeitin (TPO) receptor agonist, if approved, would be the first oral short-term treatment of previously treated patients with chronic ITP to increase platelet counts and reduce or prevent bleeding.

In the pivotal studies, the most common adverse events observed in patients taking eltrombopag were headache, nasopharyngitis, and nausea.

About the data

The NDA submission is supported by the largest database of clinical trial information on investigational therapies for chronic ITP patients. Two pivotal trials (one Phase III trial and one Phase II trial), were submitted to support the filing.

About ITP

Chronic ITP is a disorder marked by increased platelet destruction and/or inadequate platelet production in the blood, which causes an increased risk of bruising and bleeding.(2,3) There are estimated to be approximately 60,000 individuals diagnosed with chronic ITP in the U.S.(4) People with chronic ITP often bleed from small blood vessels causing bruises, nosebleeds or even fatal gastrointestinal or intra cerebral bleeds, although these are rare.(3)

About Eltrombopag

Eltrombopag is an oral, non-peptide thrombopoietin receptor agonist that has been shown in pre-clinical research and clinical trials to stimulate the proliferation and differentiation of megakaryocytes, the bone marrow cells that give rise to blood platelets. Eltrombopag was discovered as a result of research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals (Nasdaq: