Novartis has received approval from the US Food and Drug Administration (FDA) for its Promacta (eltrombopag) to treat children six years and older with chronic immune thrombocytopenia (ITP), a rare type of blood cancer.
In 2008, FDA first approved Promacta for use in adult patients with the same condition.
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Promacta is a once-daily oral thrombopoietin (TPO) receptor agonist, which works by accelerating stimulation and differentiation of megakaryocytes from bone marrow stem cells to increase platelet production.
Novartis oncology president Bruno Strigini said: "FDA approval of Promacta for children with chronic ITP, a rare and potentially serious blood disorder, gives new hope to patients and their families.
"All patients are important, but when we can help children, we are especially gratified. This approval underscores our expertise in benign hematologic disease and our commitment to provide treatments for rare diseases."
FDA approval was based on data from two double-blind and placebo-controlled studies, including the largest Phase III clinical trial in this patient population.
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By GlobalDataAccording to the firm, Promacta significantly increased and sustained platelet counts among some paediatric patients with chronic ITP, and some patients taking concomitant ITP medications have been able to reduce or discontinue their use of these medications, primarily corticosteroids.
PETIT was a Phase II, multi-centre and three-part study designed to investigate the efficacy, safety and tolerability of Promacta in paediatric patients with previously treated chronic ITP.
PETIT2 was a Phase III, multi-centre and two-part study designed to investigate the efficacy, safety and tolerability of Promacta in paediatric patients with previously treated chronic ITP.
Novartis said that safety was consistent with the known safety profile of Promacta in chronic ITP in adults and subjects in both studies.
The blood cancer drug is marketed under the brand name Promacta in the US, and as the Revolade in most countries outside the US.
Image: Novartis AG headquarters in Basel. Photo: courtesy of Andrew.
