The U.S. Food and Drug Administration (FDA) has granted approval for the first drug designed to slow muscle mass loss in patients with spinal muscular atrophy.
This drug is called atigeprostimab (Isembyld). It is a monoclonal antibody that targets precursors of myostatin protein, which regulates skeletal muscle development.
Atigeprostimab is developed for use in addition to existing therapy aimed at correcting the splicing of the SMN2 gene. The goal of this supplementary therapy is to ensure the production of the normal form of the SMN protein, which is achieved with drugs such as nusinersen and risdiplam.
Although gene splicing correction therapy significantly improves the condition of people with spinal muscular atrophy, they may still have motor limitations of varying severity. This is what atigeprostimab is intended to compensate for.
The drug is prescribed to patients starting from the age of two. Administration is carried out via intravenous infusion at a dosage of 10 milligrams per kilogram of body weight every four weeks.