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news.Swiss drugmaker Novartis has received approval from the US Food and Drug Administration (FDA) for Promacta (eltrombopag) to treat children aged six years and older with chronic immune thrombocytopenia (ITP), a rare blood disorder.
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The drug is approved for these children who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.
Earlier in 2008, the FDA approved Promacta for use in adult patients with the same condition.
Promacta is a once-daily oral thrombopoietin (TPO) receptor agonist that works by inducing stimulation and differentiation of megakaryocytes from bone marrow stem cells to increase platelet production.
Each year, ITP affects as many as five in 100,000 children and is characterized by a low platelet count.
The approval was based on data from two double-blind, placebo-controlled trials (PETIT and PETIT2), including the largest Phase III clinical trial in this patient population.
These trials showed that 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.
Weill Cornell Medical College professor of pediatrics, of pediatrics in obstetrics and gynecology and of pediatrics in medicine 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 of eltrombopag for children six years and older 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."
According to the company, Promacta should be used only in those whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.
PETIT was a Phase II, multi-center, three-part trial and PETIT2 was a Phase III, multi-center, two-part trial both designed to evaluate the efficacy, safety and tolerability of Promacta in pediatric patients with previously treated chronic ITP.
Image: Promacta is approved to treat children with chronic immune thrombocytopenia. Photo: courtesy of Michelle Meiklejohn/ freedigitalphotos.net.