Clinical Trials

The drug is an antibody that very specifically targets the latent (or inactive) form of an important protein called myostatin. Myostatin is a member of the TGFβ superfamily of growth factors. It is expressed primarily in skeletal muscle cells to inhibit muscle growth and works in concert with other growth factors and hormones to maintain appropriate muscle mass. When injected into the bloodstream, apitegromab selectively binds to latent myostatin which prevents the conversion of myostatin to its active form in muscle tissues, thereby allowing muscle growth and strength.

The European Medicines Agency (EMA) granted Priority Medicines (PRIME) Orphan Medicinal Product Designation to apitegromab for the treatment of SMA and the U.S. Food and Drug Administration (FDA) has granted it Orphan Drug Designation (ODD).