Amgen received approval from the FDA for the supplemental Biologics License Application (sBLA) for Nplate (romiplostim) for the treatment of pediatric patients one year of age and older with immune thrombocytopenia (ITP) for at least six months who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. 

The approval was based on two placebo-controlled studies evaluating the safety and efficacy of Nplate in pediatric patients. In the Phase III study, rates of overall platelet response were increased with the Nplate group (71 percent) compared with placebo (20 percent). Additionally, durable platelet response occurred more frequently with Nplate (52 percent) compared with placebo (10 percent). In the two placebo-controlled trials, adverse reactions with an incidence of > 25 percent in the Nplate arm were contusion, upper respiratory tract infection and oropharyngeal pain. 

ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood and impaired platelet production.In the U.S., the estimated prevalence of ITP in children is 5.3 per 100,000 children annually.

“This approval underscores our long-standing commitment to making a positive impact on the lives of patients with rare and difficult-to-treat hematological disorders,” said David M. Reese, M.D., executive vice president of R&D at Amgen. “In the 10 years since the FDA approved Nplate as the first platelet booster for adult patients with chronic ITP, it has made a difference in the lives of thousands of adults, and we’re proud to bring this treatment option to children who need it most.”