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Ramatercept is a recombinant fusion protein drug, also known as ACE-031, designed to inhibit myostatin and related ligands by acting as a soluble activin receptor type IIB (ActRIIB) antagonist. It functions by binding to myostatin (growth/differentiation factor 8, GDF8) and other negative regulators of muscle mass, thereby blocking their inhibitory effects on muscle growth. This mechanism promotes increased muscle mass and has been investigated for the treatment of muscle-wasting diseases such as Duchenne muscular dystrophy and other muscular atrophies. Ramatercept was initially developed by Acceleron Pharma and later involved Shire in its development; however, clinical development was discontinued due to safety concerns[1][3][5][6][8].
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