US regulators have this week approved Amgen’s supplemental Biologics License Application for Nplate for treating paediatric patients with immune thrombocytopenia (ITP).
“Today’s approval underscores our long-standing commitment to making a positive impact on the lives of patients with rare and difficult-to-treat haematological disorders,” said David M. Reese, Executive Vice President of Research and Development 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.”
The approval was based on two placebo-controlled studies – Phase 3 and Phase 1/2 – evaluating the safety and efficacy of Nplate in paediatric patients.
In the study – published in The Lancet – rates of overall platelet response were increased with the Nplate group (71%) compared with placebo (20%).
Additionally, durable platelet response occurred more frequently with Nplate (52%) compared with placebo (10%).
In the two placebo-controlled trials, adverse reactions with an incidence of > 25% in the Nplate arm were contusion, upper respiratory tract infection and oropharyngeal pain.