Romiplostim
[CAS# 267639-76-9]

Identification

• Classification: Biochemical >> Inhibitor
• Name: Romiplostim
• Protein Sequence: Thrombopoietin receptor binding domain amino acid sequence:; IEGPTLRQWLAARA; ; Amino acid sequence for Fc fusion compound:; MDKTHTCPPCPAPELLGGPSVFLFPPKPKDTLMISRTPEVTCVVVDVSHEDPEVKFNWYV; DGVEVHNAKTKPREEQYNSTYRVVSVLTVLHQDWLNGKEYKCKVSNKALPAPIEKTISKA; KGQPREPQVYTLPPSRDELTKNQVSLTCLVKGFYPSDIAVEWESNGQPENNYKTTPPVLD; SDGSFFLYSKLTVDKSRWQQGNVFSCSVMHEALHNHYTQKSLSLSPGKGGGGGIEGPTLR; QWLAARAGGGGGGGGIEGPTLRQWLAARA
• Molecular Formula: C₂₆₃₄H₄₀₈₆N₇₂₂O₇₉₀S₁₈
• Molecular Weight: 59084.16
• CAS Registry Number: 267639-76-9

Discovory and Applicatios

Romiplostim is a synthetic protein used to treat thrombocytopenia, a condition characterized by abnormally low platelet counts, primarily in individuals with chronic immune thrombocytopenia (ITP). It belongs to the class of thrombopoietin receptor agonists and is designed to mimic the natural hormone thrombopoietin, stimulating the production of platelets in the bone marrow.

Romiplostim was developed using advanced protein engineering techniques. Its discovery in the early 2000s emerged from efforts to create novel therapeutics for patients with platelet disorders, especially those unresponsive to traditional treatments like corticosteroids or immunoglobulins. The FDA approved Romiplostim in 2008 for use in patients with chronic ITP.

Romiplostim is structurally unique compared to other drugs in its class. It consists of an Fc-peptide fusion protein, also known as a "peptibody," where the peptide domain binds to the thrombopoietin receptor, and the Fc domain prolongs the drug’s half-life, allowing for weekly subcutaneous administration. This design minimizes immunogenicity compared to recombinant human thrombopoietin.

Clinically, Romiplostim is used in adult and pediatric patients with chronic ITP to boost platelet counts and reduce the risk of bleeding. Its effectiveness has been demonstrated in multiple clinical trials, showing improved platelet levels and reduced bleeding events. It is especially valuable for patients who do not respond to other therapies.

In addition to ITP, Romiplostim is being explored for potential applications in other platelet-related disorders, such as chemotherapy-induced thrombocytopenia and aplastic anemia. Its mechanism of stimulating platelet production holds promise for broader therapeutic uses in conditions where thrombopoiesis is impaired.