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Amgen’s drug reduces rates of bleeding in rare childhood disorder

Published on 21/04/16 at 09:01am

Amgen (NASDAQ: AMGN) has announced the results of a Phase III trial, published in The Lancet, which shows that Nplate (romiplostim) reduced rates of bleeding by more than 30% in children affected by rare blood disorder, symptomatic immune thrombocytopenia (ITP).

ITP is a serious autoimmune disease that is characterised by low platelet counts in the blood and impaired platelet production. In the US, it is estimated that it affects 5 in every 1,000 children each year.

The main goal of developing treaments for children with ITP is to promote a platelet count that maintains appropriate control of bleeding, improve symtpoms and increase the number of platelets.

The study met the primary endpoint of durable platelet response and showed that children who were treated with Nplate had increased rates of overall platelet response, and patients who responded to Nplate maintained consistently elevated platelet counts.

Sean E. Harper, executive VP of research and development at Amgen, says: “Nplate helps bone marrow produce more platelets, which in turn helps prevent bruising and bleeding which is important for children faced with this condition. These data are important in understanding how Nplate may play a role in helping children manage the disease. We will work with regulatory authorities towards an approval for Nplate for paediatric patients.”

Sean Murray

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