FDA approves extension of use of Promacta (eltrombopag) to children for thrombocytopenia- Novartis

Novartis announced that the FDA has approved Promacta (eltrombopag) for the treatment of children six years and older with chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. Promacta was approved by the FDA in 2008 for use in adult patients with the same condition.

ITP affects as many as 5 in 100,000 children each year and is characterized by a low platelet count. Up to 30% of affected children experience persistent disease for more than 6 months and are diagnosed with chronic ITP. Pediatric patients with chronic ITP are at ongoing risk of significant bleeding.

The approval of Promacta was based on data from two double-blind, placebo-controlled trials, including the largest Phase III clinical trial in this patient population. Treatment with Promacta significantly increased and sustained platelet counts among some pediatric patients with chronic ITP, and some patients taking concomitant ITP medications were able to reduce or discontinue their use of these medications, primarily corticosteroids. Promacta should be used only in those whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

Comment: Promacta was one of the oncology drugs transferred to Novartis by GSK in exchange for vaccines from Novartis.