Rare Blood Disorder Drug Approved for Kids

The FDA has approved Novartis’s eltrombopag (Promacta) to treat a rare, chronic blood disorder in children.

Promacta is now indicated for both adults and children aged 6 years and older with chronic immune thrombocytopenia (ITP) who have not sufficiently responded to corticosteroids, immunoglobulins, or splenectomy.

ITP is characterized by a low platelet count and affects as many as 5 in 100,000 children each year. As many as 30% of these children experience the disease for more than 6 months and are diagnosed with chronic ITP, putting them at ongoing risk for significant bleeding.

“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 6 years and older particularly important,” said James B. Bussel, MD, lead investigator of a clinical trial on the drug, in a press release. “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.”

Promacta’s expanded approval was based on data from 2 clinical studies in which the drug significantly increased and sustained platelet counts.

It remains the first and only oral thrombopoietin-receptor agonist that increases platelet production.

Promacta should be used only in those whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.