Amgen has 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 (for example,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 I/II placebo-controlled study, and two long-term safety and efficacy studies) and one ongoing long-term safety and efficacy study.
“Children with ITP are at risk for serious bleeding events due to low platelet counts, and currently, limited therapeutic options are available to treat this rare disease,” said Sean E Harper, M.D., executive vice president of Research and Development at Amgen. “We are pleased by today’s positive CHMP opinion and look forward to working with European regulatory authorities to deliver on our commitment to bring Nplate to paediatric patients with ITP who are suffering from thrombocytopenia.”
ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood (a condition known as thrombocytopenia) and impaired platelet production.1,2 European studies report incidence rates in children between four and five cases per 100,000 children each year.3 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.1,4
The CHMP positive opinion will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, a centralised marketing authorisation will be granted that will be valid in the 28 countries that are members of the EU. Norway, Iceland and Liechtenstein, as members of the European Economic Area (EEA), will take corresponding decisions on the basis of the decision of the EC.
- Patient. Immune Thrombocytopenia. http://patient.info/health/immune-thrombocytopenia-leaflet. Accessed Aug. 29, 2017.
- Cines DB et al. Immune thrombocytopenic purpura. N Engl J Med. 2002;346:995-1008.
- Segal JB, Powe NR. Prevalence of immune thrombocytopenia: analyses of administrative data. J Thromb Haemost. 2006;4(11):2377-83.
- US National Institutes of Health. Immune Thrombocytopenia. http://www.nhlbi.nih.gov/book/export/html/4917. Accessed Aug. 29, 2017.