Amgen announced that the European Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending marketing authorisation for Nplate(TM) (romiplostim) in the European Union (EU).
The CHMP recommends Nplate for adult chronic immune (idiopathic) thrombocytopenia purpura (ITP) splenectomised patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Nplate may be considered as second line treatment for adult non-splenectomised patients where surgery is contra-indicated.
"Nplate will address an unmet medical need for thousands of patients in the European Union as it is a unique treatment option that increases platelet production and avoids immune suppression in adult chronic ITP patients," said Willard Dere, M.D., senior vice president and international chief medical officer at Amgen.
The novel peptibody technology upon which romiplostim is based represents a promising new approach for treating adult patients with chronic ITP, an autoimmune disorder affecting an estimated 50,000 people in the EU, which can lead to serious bleeding events that can be potentially life threatening.
Nplate, a thrombopoietin (TPO) mimetic, is a novel engineered therapeutic fusion protein with attributes of both peptides and antibodies, but is distinct from each. Nplate works similarly to TPO, a natural protein in the body. Nplate stimulates the TPO receptor, which is necessary for growth and maturation of bone marrow cells that produce platelets.
The CHMP positive opinion is based on data from two separate placebo-controlled Phase 3 studies, demonstrating that platelet counts were raised and sustained in 83 percent of patients for both splenectomised and non-splenectomised groups when treated with Nplate. Additionally, patients treated with Nplate were able to reduce or discontinue concomitant ITP and emergency medications which are often not well tolerated or whose effects are transient (i.e. corticosteroids, IVIG, Win-Rho Anti-D therapy).
Upon completion of the Phase 3 studies almost 90 percent of patients elected to subsequently enroll into the romiplostim long term extension study which demonstrated that, after three years, Nplate continued to effectively increase and sustain platelet counts. In this open label long term extension study some patients were treated for over 156 weeks and in the interim analysis the median treatment duration in this study is 65 weeks.