First medicine for spinal muscular atrophy is under accelerated assessment
Orphan medicine Spinraza recommended by CHMP under accelerated assessment. The European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union (EU) for Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Spinraza is to be given by lumbar puncture injection into the fluid surrounding the spinal cord once every four months.
