Data from a late-stage trial testing Amgen’s Nplate in children with symptomatic immune thrombocytopenia (ITP) shows that the drug induced a durable platelet response, raising hopes for a new treatment option to help reduce bleeds in patients.
Findings from the Phase III study, published in The Lancet, show that 52 percent of patients receiving Nplate (romiplostim) achieved a durable platelet response compared with 10 percent of those in the placebo arm, meeting the primary endpoint.
“Children with ITP are at risk for serious bleeding events due to low platelet counts, which can be very frightening for these children and their parents,” said Michael Tarantino, professor of Pediatrics and Medicine at the University of Illinois College of Medicine-Peoria.
Those who were treated with Nplate had increased rates of overall platelet response, and patients who responded to Nplate maintained consistently elevated platelet counts, the firm noted.
“The results of this study suggest that romiplostim could reduce the frequency and severity of bleeding events for children suffering from symptomatic ITP, thus providing them with another potential treatment option,” Professor Tarantino noted.
The most frequently reported adverse events included contusion, epistaxis, headache and upper respiratory tract infections, but the overall safety profile observed in the Nplate arm was similar to the drug’s known safety profile.
Nplate is a thrombopoietin receptor agonist indicated for the treatment of low blood platelet counts in adults with ITP - a rare, serious autoimmune disease - who had an insufficient response to other medicines or surgery. Amgen said it will now work with regulatory authorities towards an approval for paediatric patients.