FDA approves Amgen's Nplate for certain paediatric patients with immune thrombocytopaenia

Amgen on Friday announced that the FDA expanded the indication for Nplate (romiplostim) to include its use in paediatric patients aged one year and older with immune thrombocytopaenia (ITP) for at least six months who experienced an insufficient response to corticosteroids, immunoglobulins or splenectomy. Amgen executive vice president of R&D David Reese stated "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."  

The approval was supported by efficacy and safety results from Phase III and Phase I/II studies involving paediatric patients aged one year and older with ITP for at least 6 months duration. In study data unveiled in 2016, Nplate was associated with an overall plate response rate of 71 percent, versus 20 percent for placebo. Additionally, the durable platelet response rates in the Nplate and placebo groups were 52 percent and 10 percent, respectively.  

The thrombopoietin receptor agonist was approved in 2008 in the US for the treatment of chronic immune thrombocytopaenic purpura in patients who do not sufficiently respond to existing therapeutics.