GlaxoSmithKline (GSK) announced 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.
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 an oral, non-peptide, thrombopoietin receptor agonist. It stimulates the proliferation and differentiation of megakaryoctes, resulting in an increase in platelet counts.
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.