FDA approves Promacta for pediatric patients with chronic immune thrombocytopenia
The FDA announced the approval of eltrombopag for use in children aged 6 years or older with chronic immune thrombocytopenia who experienced an insufficient response to corticosteroids, immunoglobulins or splenectomy, according to a press release.
Eltrombopag (Promacta, Novartis) is a once-daily oral thrombopoietin receptor agonist that increases platelet production in patients with chronic immune thrombocytopenia, according to a press release. The FDA approved eltrombopag for use in adults in 2008.
Immune thrombocytopenia affects up to five in 100,000 children annually. Approximately 30% of affected children experience persistent disease for more than 6 months and are diagnosed with chronic immune thrombocytopenia. Pediatric patients face a risk for significant bleeding.
The most common treatments for chronic immune thrombocytopenia — including corticosteroids and IV immunoglobulin — often result in intolerable adverse events for pediatric patients, according to the press release.
The FDA based its decision to approve eltrombopag in children in part on data from two double-blind, controlled trials, including the largest phase 3 clinical trial conducted in this patient population. Results showed eltrombopag significantly increased and sustained platelet counts among pediatric patients. Further, eltrombopag therapy allowed some patients taking concomitant medications — particularly corticosteroids — to reduce or discontinue their use of these medications.
The most common adverse events associated with the use of eltrombopag in children are upper respiratory tract infections, nasopharyngitis, rhinitis, abdominal pain, cough, inflammation in the throat or mouth, toothache, abnormal liver function tests, diarrhea, rash and vitamin D deficiency.
Eltrombopag should be used only by patients whose degree of thrombocytopenia and clinical condition increase the risk for bleeding, according to the press release.
“[The] FDA approval of Promacta for children with chronic immune thrombocytopenia, a rare and potential serious blood disorder, gives new hope to patients and their families,” Bruno Strigini, PharmD, MBA, president of Novartis Oncology, said in a press release. “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.”