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Rindopepimut is an injectable peptide cancer vaccine designed to target the epidermal growth factor receptor variant III (EGFRvIII), a tumor-specific mutation found in approximately 25–30% of glioblastoma cases. The drug consists of a synthetic peptide that mimics the EGFRvIII mutation, conjugated to keyhole limpet hemocyanin (KLH) as an immunogenic carrier. By presenting this neoantigen, rindopepimut stimulates the immune system—particularly inducing EGFRvIII-specific antibody responses and antibody-dependent cellular cytotoxicity—to selectively attack and eliminate tumor cells expressing EGFRvIII. It was primarily developed for adult patients with newly diagnosed or recurrent glioblastoma multiforme (GBM) who test positive for EGFRvIII. Despite promising results in early-phase trials, pivotal phase III studies failed to show improved overall survival, leading to discontinuation of its development for GBM[1][2][3][4][5][7][8].
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