European Medical Committee Endorses Nplate

The European Committee for Medicinal Products for Human Use (CHMP) has recommended Nplate for marketing in the European Union. Nplate (romiplostim) is currently the only long term drug on the market for treating chronic Idiopathic thrombocytopenic purpura (ITP), a condition of having low blood platelet counts.

Nplate is a fusion protein that contains characteristics of both antibodies and peptides. It works by stimulating the TPO receptor, causing bone marrow cells to grow, which make blood platelets.

There are an estimated 50,000 ITP patients in Europe, however not all of them are being recommended for Nplate. Only the patients who aren’t responding to current treatments are suggested to try Nplate. This could still be several thousands of people.

The CHMP is basing its recommendation on two different phase 3 studies where platelet counts were raised by 83% in patients from both splenectomised and non-splenectomised groups.

Before considering Nplate, patients should look at the potential side effects. Because Nplate works by stimulating bone marrow growth, there is the possibility of getting bone marrow fibrosis. Patients may also experience lower platelet counts after discontinuing Nplate compared to when started taking Nplate. Another risk is blood clot formation; as platelet counts increase, there may be a higher risk for strokes or heart attacks caused by blood clots.

What is your opinion? Please share your experiences and opinions about Nplate and ITP.