CHMP recommends extension of Nplate indication to children for thrombocytopenic purpura. -Amgen.

Amgen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion to expand the current indication for Nplate (romiplostim) to include the treatment of chronic immune (idiopathic) thrombocytopenic purpura (ITP) for patients one year of age and older who are refractory to other treatments (e.g., corticosteroids, immunoglobulins).

The positive CHMP opinion was based on five studies evaluating the safety and efficacy of Nplate in children with ITP, including four completed studies (a Phase III, a Phase 1/II placebo-controlled study, and two long-term safety and efficacy studies) and one ongoing long-term safety and efficacy study.

ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood (a condition known as thrombocytopenia) and impaired platelet production. European studies report incidence rates in children between four and five cases per 100,000 children each year. The treatment goal for children with ITP is to promote a platelet count that maintains appropriate control of bleeding, improve symptoms and increase the number of platelets.