The Food and Drug Administration has extended its approval of Promacta (eltrombopag) to include pediatric patients with chronic immune thrombocytopenic purpura, the agency announced Aug. 24.
Approved in 2008 for adults with immune thrombocytopenic purpura (ITP), eltrombopag is now approved for the treatment of the rare blood disorder in patients aged 1 year and older. The drug may be used in children who have not responded to other ITP medications or spleen surgery, the FDA said in a statement.
Eltrombopag’s efficacy and safety in children aged 1-17 years was established in two placebo-controlled trials comprising 159 patients. Findings from the first trial found that over 7 weeks, 62% of patients given eltrombopag had improved platelet counts without rescue therapy between weeks 1 and 6, compared with 32% in the placebo group.
In the second trial, which lasted 13 weeks, 41% of patients taking eltrombopag experienced increased platelet counts for at least 6 out of 8 weeks between weeks 5 and 12, compared with 3% of patients in the placebo group, the FDA reported.
“In both trials, patients taking Promacta also had less need for other treatments to increase their platelet counts, such as corticosteroids or platelet transfusions,” the FDA said. “Among patients taking one or more ITP medications at the start of the trials, about half were able to reduce or discontinue their use of these medications, primarily corticosteroids.”
Eltrombopag may be taken once daily in tablet form, or as a powder mixed with liquid for children aged 1-5 years. It should be used only in ITP patients with an increased risk of bleeding.
The most common side effects in children were infections of the upper respiratory tract or nose and throat, diarrhea, abdominal pain, rash, and increase in liver enzymes.
Promacta is manufactured by Novartis in East Hanover, N.J.