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Apitegromab is a fully human monoclonal antibody developed by Scholar Rock that selectively inhibits the activation of myostatin (GDF8). Unlike traditional myostatin inhibitors that target the mature growth factor or its receptor, apitegromab binds to the pro- and latent forms of myostatin, preventing the release of the active mature protein in skeletal muscle. This high selectivity is designed to avoid the inhibition of related TGFβ superfamily members like GDF11 and Activin A, potentially improving the safety profile. The drug is primarily being developed as a muscle-targeted therapy for spinal muscular atrophy (SMA) to improve motor function in patients already receiving SMN-targeted treatments. Additionally, it is being evaluated for its ability to preserve lean muscle mass in individuals with obesity who are undergoing weight loss treatment with GLP-1 receptor agonists.
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