FDA has approved eltrombopag (Promacta, Novartis) for the treatment of children aged 6 years and older with chronic immune thrombocytopenia.
FDA has approved eltrombopag (Promacta, Novartis) for the treatment of children aged 6 years and older with chronic immune thrombocytopenia (cITP).
Immune thrombocytopenia (ITP) is a bleeding disorder in which a person’s immune system attacks their own platelets. Due to a low number of platelets, the blood does not clot as it should and consequently, people with ITP can bruise of bleed more easily than normal. ITP affects as many as 5 in 100,000 children each year, and up to 30 percent of those affected experience persistent disease for more than 6 months and are diagnosed with chronic ITP. Patients with chronic ITP are at an ongoing risk of significant bleeding.
Promacta is a once-daily thrombopoietin receptor agonist that interacts with the thrombopoietin receptor to increase platelet production. It is now approved to treat children aged 6 years and older with chronic ITP, when corticosteroids, immunoglobulins, or splenectomy have not been successful. It was approved in 2008 for use in adults with the same condition.
“[The] FDA approval of Promacta for children with chronic ITP, a rare and potentially serious blood disorder, gives new hope to patients and their families,” said Bruno Strigini, president, Novartis Oncology. “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.”
The drug's approval was based on 2 double-blind, placebo-controlled trials, including the largest phase 3 clinical trial in this patient population. Results from the PETIT and PETIT2 trials showed that Promacta significantly increased and sustained platelet count among some pediatric patients with chronic ITP, and some patients who were taking other ITP medications were able to reduce or discontinue the use of these medications.
It is not known if Promacta is safe and effective in ITP patients younger than age 6 years. Separate applications were submitted to FDA earlier this year to include chronic ITP patients aged 1 year and older. Upper respiratory tract infection, nasopharyngitis, and rhinitis were the most common adverse reactions associated with the use of Promacta in chronic ITP patients aged 6 years and older.