Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of a Romiplostim Biosimilar in Chronic Refractory Immune Thrombocytopenic Purpura (ITP) Patients

Romiplostim (Nplate (R), Amgen Inc.) is an orphan drug approved for the treatment of chronic refractory immune thrombolytic purpura (ITP) in adult and pediatric patients. Limited availability of pharmacokinetic (PK) data and large inter- and intra-subject variability in PK and platelet response is a challenge in the clinical development of a romiplostim biosimilar. We compared pharmacokinetics (PK), pharmacodynamics (PD)/efficacy, and safety of a romiplostim biosimilar with Nplate in 24 patients with ITP following a single 3 mu g/kg dose, and assessed efficacy of the romiplostim biosimilar at a titrated dose range of 1-5 mu g/kg in 50 patients with ITP. The PK of the romiplostim biosimilar did not differ compared to the PK of Nplate, and PD/efficacy responses were similar between the products following the single dose. The romiplostim biosimilar showed historically comparable PD/efficacy with Nplate over 8 weeks when treated at the titrated dose range. It was well tolerated in both the studies.