February 26, 2021 – EMA has recommended granting a marketing authorisation in the European Union (EU) for the first treatment that can be given orally to patients with certain types of spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

Spinal muscular atrophy is an inherited disease usually diagnosed in the first year of life that affects the motor neurons (neurons from the brain and spinal cord that control muscle movements). Patients with the disease lack a protein called ‘survival motor neuron’ (SMN), which causes the motor neurons to deteriorate and eventually die. This is a long-term debilitating and life-threatening disease because it causes breathing problems and muscle wasting that worsens over time.