Long term safety of Revolade (eltrombopag) in adults with chronic immune (idiopathic) thrombocytopenia.- Novartis

Novartis announced data from the largest study of its kind confirming the long-term safety profile of Revolade (eltrombopag) in adults with chronic immune (idiopathic) thrombocytopenia (ITP), with data for up to 6 years in some patients (median exposure was 2.4 years). Additional data from the study will also be presented that showed long-term oral administration of Revolade was effective in increasing and maintaining platelet counts in adult patients who had their spleens removed (splenectomized) as well as those who did not (non-splenectomized). The final results of the study and sub-analysis will be presented at the 21st Congress of the European Hematology Association (EHA) in Copenhagen, Denmark.

ITP is a rare and potentially serious blood disorder where the blood does not clot as it should due to a low number of platelets. As a result, patients experience bruising, bleeding and, in some cases, serious hemorrhage that can be fatal. ITP may also affect a patient's quality of life, as it is often associated with fatigue and depression as well as a fear of bleeding that may limit everyday activities.

The safety profile of Revolade seen in the EXTEND trial is consistent with that observed in the pivotal 24-week Phase III RAISE study. Long-term use of Revolade was not associated with a clinically relevant increase in bone marrow reticulin or collagen fibers. The most common adverse events were headache (28%), nasopharyngitis (25%), upper respiratory tract infection (23%), and fatigue (17%). The efficacy results of EXTEND demonstrated that median platelet counts were elevated to more than =50 � 109/L within two weeks of Revolade treatment, with median platelet counts more than 50 � 109/L maintained for more than four years. Post-baseline, overall bleeding rates declined and the majority of bleeding that occurred during more than six years of the study was Grade 1 according to the World Health Organization bleeding scale. In addition, 91.4% (276/302) of patients achieved platelet counts more than =30 � 109/L without rescue treatment, and 85.8% (259/302) achieved platelet counts more than =50 � 109/L without rescue treatment.

Comment: Revolade is approved in more than 100 countries for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an inadequate response or are intolerant to other treatments. Eltrombopag (marketed as Promacta in the US) is approved in the EU and the US for patients one year and older with chronic ITP who have had an insufficient response to other treatments. Revolade is also approved in 45 countries for the treatment of patients with severe aplastic anemia (SAA) who are refractory to other treatments, and in more than 50 countries for the treatment of thrombocytopenia in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy.