EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma (onasemnogene abeparvovec) to treat babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Spinal muscular atrophy is usually diagnosed in the first year of life. Most patients with severe SMA do not survive early childhood. Patients with the disease cannot produce sufficient amounts of a protein called ‘survival motor neuron’ (SMN), which is essential for the normal functioning and survival of motor neurons (nerves from the brain and spinal cord that control muscle movements). Without this protein, the motor neurons deteriorate and eventually die. This causes the muscles to fall into disuse, leading to muscle wasting (atrophy) and weakness. EMA’s recommendation for conditional marketing authorisation is based on the preliminary results of one completed clinical trial and three supporting studies in patients with spinal muscular atrophy with different stages of disease severity. These included genetically diagnosed and pre-symptomatic patients.
The clinical trial providing the main body of data for the assessment of Zolgensma was conducted in 22 patients who were less than six months of age at the time of the gene replacement therapy with Zolgensma. Currently, treatment with Zolgensma should only be administered once in suitable clinical centres under the supervision of a physician experienced in the management of patients with SMA.
Further information is available here.