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Novartis gets FDA nod for rare blood disorder drug

Promacta will provide a treatment option for children over 6 years old

Novartis building 

The FDA has approved Novartis' Promacta for the treatment of children over six with chronic immune thrombocytopenia (ITP), seven years after it was given the nod for adults.

Promacta (eltrombopag) will be a treatment option for child patients who have had an insufficient response to corticosteroids, immunoglobins or splenectomy following its approval for adults in 2008.

Bruno Strigini, president of Novartis Oncology, commented: “Today's 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.”

Promacta is a once-daily oral thrombopoietin (TPO) receptor agonist that works by inducing stimulation and differentiation of large cells from bone marrow stem cells to increase platelet production.

The approval was based on data from two double-blind, placebo-controlled trials that showed a significant increased and sustained platelet count among some paediatric patients with chronic ITP, while some patients taking concomitant ITP medications were also able to reduce of cease the use of medications.

James Bussel, a professor of pediatrics, of pediatrics in obstetrics and gynecology and of pediatrics in medicine at Weill Cornell Medical College, and lead study investigator, added: “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.”

Article by
Kirstie Pickering

17th June 2015

From: Sales



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