FDA Approves Novartis’ Promacta to Treat Paediatric Patients
Novartis has received approval from the FDA for Promacta to treat children aged six years and older with chronic immune thrombocytopenia (ITP). ITP is a rare blood disorder that affects as many as five in 100,000 children every year and is characterised by a low platelet count. The drug has been approved for children with ITP who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. In 2008, Promacta had been previously approved for use in adults suffering from the same disorder.
Promacta is a once-daily oral thrombopoietin (TPO) receptor agonist that induces stimulation and differentiation of megakaryocytes from bone marrow stem cells resulting in increased platelet production. According to Novartis, Promacta should be used only in those whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.
Results from Phase III trials show that treatment with Promacta drastically increases and sustains platelet counts among some paediatric patients with chronic ITP. It also allowed some patients taking concomitant ITP medications to reduce or discontinue their use of these medication, in particular corticosteroids.
The approval was based on data from two double-blind, placebo-controlled trials (PETIT and PETIT2). PETIT was a Phase II, multi-center, three-part trial and PETIT2 was a Phase III, multi-center, two-part trial. Both were designed to evaluate the efficacy, safety and tolerability of Promacta in paediatric patients with previously treated chronic ITP.
Medical College professor of paediatrics, Weill Cornell, and lead study investigator of the PETIT study James Bussel said: “Young patients with chronic ITP who have either an insufficient response to or side effects from standard therapies have limited treatment options, making this FDA approval particularly important. Through the eltrombopag studies, one of which is the largest randomized trial ever performed in children with chronic ITP, we discovered that Promacta – a treatment that can be taken once daily by mouth and shown to be well tolerated – can manage this disorder and help these young patients.”