FDA expands pediatric age indication for Promacta

August 26, 2015

FDA has expanded approval of eltrombopag (Promacta, Novartis) to treat low blood platelet count in pediatric patients with chronic immune thrombocytopenic purpura.

FDA has expanded approval of eltrombopag (Promacta, Novartis) to treat children aged 1 year and older with chronic immune thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.

ITP is a blood disorder characterized by unusually low levels of platelets, which can lead to easy or excessive bruising and bleeding. People who have ITP often have purple bruises or tiny red or purple dots on the skin caused by bleeding under the skin. In most cases, an autoimmune response, in which a person’s immune system attacks its own platelets, is thought to cause ITP. Chronic ITP, which affects approximately 1 in 4 children with ITP, is defined as ongoing disease more than 12 months after diagnosis.

Promacta was first approved in 2008 to treat low platelet counts in adult patients with chronic ITP.

Related:Promacta stimulates platelet production in cITP pediatric patients

Promacta acts as a thrombopoietin (TPO) receptor agonist to increase platelet counts in order to reduce the risk for bleeding. It should only be used in patients whose degree of thrombocytopenia and overall clinical condition increases the risk for bleeding. Promacta is available as a tablet taken once-daily or as an oral suspension for children aged 1 to 5 years.

"It's challenging and often very emotional for parents of a baby or toddler affected by a rare condition to manage their child's disease with limited treatment options," said Bruno Strigini, President, Novartis Oncology. "Today's label expansion for Promacta provides a new disease management option for families affected by chronic ITP and highlights our commitment to providing treatments for even the youngest children with rare diseases."

The expanded approval of Promacta was based on data from 2 double-blind, placebo-controlled trials; PETIT and PETIT2. Results of these trials demonstrated significantly increased and sustained platelet counts among pediatric patients with chronic ITP with an insufficient response to prior therapies. Some patients taking concomitant ITP medications were also able to reduce or stop their use of these medications.  

The most common side effects associated with the use of Promacta in children aged 1 year and older include upper respiratory tract infections, inflammation in the nose and throat, cough, diarrhea, abdominal pain, and runny nose. 

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