The Food and Drug Administration has approved Promacta for the treatment of chronic immune thrombocytopenia in children 6 years and older, Novartis, the drug’s manufacturer, announced in a statement.
Promacta (eltrombopag) is an oral thrombopoietin receptor agonist that increases platelet production by inducing stimulation and differentiation of megakaryocytes from bone marrow stem cells. Chronic immune thrombocytopenia (ITP) patients often have low platelet counts and are at risk for significant bleeding.
Promacta was approved for adult use in 2008; the drug was evaluated for pediatric use in PETIT and PETIT2, two multiphase, double-blind placebo-controlled trials designed to evaluate the drug’s safety profile. Both studies found Promacta’s safety to be consistent with the known safety profile of Promacta in chronic ITP in adults.
Promacta’s manufacturers say the drug can be useful for chronic ITP patients who have had an insufficient response to the most commonly available and used therapies for chronic ITP, such as corticosteroids, intravenous immunoglobulin, or splenectomy. ITP affects as many as 5 in 100,000 children each year.
The most common adverse reactions to Promacta in pediatric chronic ITP patients were upper respiratory tract infection, nasopharyngitis, and rhinitis. In adults, Promacta has been associated with nausea, diarrhea, upper respiratory tract infection, and vomiting; rarer but more serious side effects include liver problems, high platelet counts, a higher risk for blood clots, and new or worsened cataracts.