ロミプロスチム（遺伝子組換え） 化学特性,用途語,生産方法
説明
Romiplostim has been developed and launched for the treatment of
thrombocytopenia in patients with ITP, an autoimmune blood disorder in
which there is autoantibody-mediated platelet destruction. Coating of
the platelets by autoantibodies results in accelerated ingestion by
macrophages. In addition to the destruction component, ITP is also
characterized by impaired platelet production. The primary physiological regulator of platelet production is thrombopoietin (TPO), a growth
factor that orchestrates its effects through the TPO (Mpl) receptor.Agonists of this receptor could, thereby, remediate the platelet production contribution of ITP. By binding to and activating the TPO receptor,
romiplostim serves as a TPO receptor agonist. As a recombinant fusion
protein, in this case coined as a peptibody, it was engineered to contain
two identical single-chain subunits, each consisting of a TPO-binding
domain linked to the C-terminus of a human IgG1 Fc domain designed to
increase the half-life of the protein. Although it binds to the TPO
receptor, romiplostim has no sequence homology to endogenous TPO,
which should mitigate the risks encountered with the first generation
predecessor, recombinant megakaryocyte growth and development
factor (MGDF); this truncated, non-glycosylated form of TPO conjugated
to PEG was halted during clinical studies because of the immunogenicity
of PEG-MGDF resulting in cross-reacting (neutralizing) antibodies
against endogenous TPO and subsequent severe, unrelenting thrombocytopenia. Romiplostim also avoids the side effects associated with the
generalized immunosuppressive agents previously employed for the
treatment of ITP and may prevent splenectomy, another common
treatment option.
Originator
Amgen (United States)
brand name
Nplate
ロミプロスチム（遺伝子組換え） 上流と下流の製品情報
原材料
準備製品