EAN 2021 | Apitegromab for the treatment of SMA

In 2021, apitegromab was granted fast track designation by the FDA for the treatment of patients with spinal muscular atrophy (SMA). Gabriele Siciliano, MD, PhD, University of Pisa, Pisa, Italy, talks on apitegromab’s mechanism of action and how it is likely to develop clinically in the coming years. Apitegromab is a monoclonal antibody directed against myostatin which prevents the conversion of latent myostatin into the active form that inhibits muscle growth. An ongoing proof-of-concept Phase II trial (NCT03921528) has demonstrated promising early signs of efficacy and safety in children and young adults with SMA types 2 and 3. Based on these finding a Phase III trial of apitegromab is planned. If successful, it has the potential to be the first muscle-directed therapy for patients with spinal muscular atrophy. This interview took place during the European Academy of Neurology 2021 congress.