, Volume 69, Issue 3, pp 307–317


  • James E. Frampton
  • Katherine A. Lyseng-Williamson
Adis Drug Profile

DOI: 10.2165/00003495-200969030-00006

Cite this article as:
Frampton, J.E. & Lyseng-Williamson, K.A. Drugs (2009) 69: 307. doi:10.2165/00003495-200969030-00006


  • ▴ Romiplostim is an Fc-peptide fusion protein (or ‘peptibody’) that stimulates megakaryopoiesis and thrombopoiesis by binding to, and activating, the thrombopoietin receptor.

  • ▴ Because it has no sequence homology to thrombopoietin, romiplostim theoretically avoids the risk of eliciting cross-reacting, neutralizing antibodies to thrombopoietin.

  • ▴ In well designed, 24-week, phase III trials, subcutaneous romiplostim was significantly more effective than placebo in achieving the primary endpoint of a protocol-defined durable platelet response in nonsplenectomized (61% vs 5%) or splenectomized (38% vs 0%) adults with chronic immune (idiopathic) thrombocytopenic purpura (ITP).

  • ▴ Romiplostim was also significantly more effective than placebo with regard to a number of secondary endpoints, including the proportions of patients with an overall (durable plus transient) platelet response or who required ITP rescue medications. The majority of romiplostim-treated patients receiving concurrent ITP drugs were able to reduce or discontinue these therapies.

  • ▴ Platelet response was maintained by most patients during longer-term treatment with romiplostim for up to 3 or 4 years in an open-label extension study.

  • ▴ Romiplostim was generally well tolerated. Almost all adverse events in the phase III studies were of mild-to-moderate intensity; most were unrelated to treatment. Longer-term treatment with romiplostim had an adverse event profile consistent with that observed in the phase III studies.

Copyright information

© Adis Data Information BV 2009

Authors and Affiliations

  • James E. Frampton
    • 1
    • 2
  • Katherine A. Lyseng-Williamson
    • 1
    • 2
  1. 1.Wolters Kluwer Health ¦ AdisMairangi Bay, North Shore 0754, AucklandNew Zealand
  2. 2.Wolters Kluwer HealthConshohockenUSA