Revolade (eltrombopag) receives positive opinion in Europe for chronic immune thrombocytopenic purpura

Issued: London UK

GlaxoSmithKline (GSK) announced today that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Revolade (eltrombopag), for the oral treatment of thrombocytopenia (reduced platelet count) in adults with the blood disorder chronic immune (idiopathic) thrombocytopenic purpura (ITP).

The CHMP has recommended the marketing authorisation of eltrombopag in the European Union for the treatment of ITP in adult patients who have had their spleen removed, and who do not respond to other treatments, such as corticosteroids and immunoglobulins therapies. Eltrombopag may also be considered as a second-line treatment for adult patients where surgery to remove their spleen is contraindicated.

“Eltrombopag is an innovative treatment for thrombocytopenia in patients with chronic ITP. This once-a-day tablet is able to stimulate platelet production and reduce the risk of bleeding in a difficult-to-treat disease,” said Paolo Paoletti, SVP and Global Head of Oncology Research and Development, GSK. “Eltrombopag is another example of our continued investment in R&D and our long-term commitment to improving the lives of patients.”    

ITP patients experience bruising and bleeding and, in some cases, serious haemorrhages, which can be fatal. ITP may also affect a patient’s quality of life, as it is often associated with fatigue and depression and a fear of bleeding may limit everyday activities.  Traditional treatments with corticosteroids, immunoglobulins or splenectomy (removal of the spleen) all have potential drawbacks for chronic treatment of ITP patients.4, 

Eltrombopag significantly increases platelet counts

The positive opinion from CHMP is based on two Phase III randomised, double-blind, placebo-controlled clinical trials (TRA100773B and RAISE TRA102537) and two open-label studies (REPEAT TRA108057 and EXTEND TRA105325) in adults who have previously received treatment for chronic ITP. The studies showed that the patients treated with eltrombopag (plus the standard of care) experienced significant increases in platelet counts, a reduction in the incidence of bleeding and an improvement in quality of life, compared with those receiving placebo (plus the standard of care). Eltrombopag treatment has also allowed patients to reduce the dose of their concomitant medications, such as steroids.

In clinical trials, eltrombopag was well-tolerated.  In some cases, nausea and vomiting were recorded in the eltrombopag group and not in the placebo group. Elevation of liver enzymes was also seen, but these were mostly mild, reversible and not accompanied by any clinically significant symptoms that would indicate impaired liver function.

Eltrombopag is the first oral platelet generator

Eltrombopag is an oral, non-peptide, thrombopoietin receptor agonist. It stimulates the proliferation and differentiation of megakaryoctes, resulting in an increase in platelet counts. Megakaryocytes are the bone marrow cells that give rise to blood platelets.

About eltrombopag

Eltrombopag was given accelerated approval by the US Food and Drug Administration (FDA) under the trade name Promacta® in November 2008, for the treatment of chronic ITP in adults who have had an insufficient response to corticosteroids, immunoglobulins or surgical removal of the spleen. Eltrombopag is also approved under the trade name Revolade in Venezuela, Kuwait, Chile and Russia. In addition, orphan designation was granted by the European Commission for eltrombopag for the treatment of ITP on 3 August 2007. Eltrombopag was discovered as a result of a research collaboration between GSK and Ligand Pharmaceuticals (NASDAQ: LGND), and developed by GSK.

About chronic ITP

Chronic ITP is a serious condition, where patients have low platelet levels in the blood. Platelets are essential to normal clotting, so patients with ITP are at increased risk of bleeding, and may develop bruises and experience nose or gum bleeds, have blood in the urine or faeces, abnormally heavy menstrual bleeding, or other types of bleeding that is difficult to stop. Although very rare in occurrence, bleeding in the brain is potentially fatal. Quality of life is adversely affected in patients with chronic ITP, with a fear of bleeding limiting patients’ daily activities. Fatigue and depression are, also, often associated with the disease.

GlaxoSmithKline – one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer.  For further information please visit www.gsk.com

Notes to editors

Revolade® and Promacta® are registered trade marks of the GlaxoSmithKline group of companies.

GlaxoSmithKline Enquiries: 

Cautionary statement regarding forward-looking statements
Under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Factors that may affect GSK' s operations are described under 'Risk Factors' in the 'Business Review' in the company' s Annual Report on Form 20-F for 2008.