taldefgrobep alfa

Taldefgrobep alfa is a fully human recombinant fusion protein and anti-myostatin adnectin designed to inhibit both myostatin and activin receptor signaling. It selectively binds to myostatin (GDF-8) and acts as a competitive inhibitor of ligands that signal through the activin II receptor, thereby blocking downstream activity that leads to muscle atrophy. The drug is engineered with a human IgG1 Fc tail for prolonged half-life, allowing subcutaneous administration. Its mechanism of action involves preventing the formation of the myostatin–activin receptor complex, which inhibits Smad2/3 phosphorylation and blocks muscle-wasting signals in skeletal muscle. Taldefgrobep alfa has been developed primarily for spinal muscular atrophy (SMA), where it aims to enhance muscle function by lowering free myostatin levels and antagonizing activin 2b receptors. It has also been investigated in Duchenne muscular dystrophy (DMD) and obesity[2][3][4][5][6][7].

Other names

Anti-myostatin adnectinMyostatin-inhibiting adnectin fusion protein