Spinraza

Spinraza (nusinersen), the first drug approved to treat children and

adults with spinal muscular atrophy (SMA), a rare and often fatal

genetic disease affecting muscle strength and movement. Spinraza is an

injection administered into the fluid surrounding the spinal cord.<p>“There

has been a long-standing need for a treatment for spinal muscular

atrophy, the most common genetic cause of death in infants, and a

disease that can affect people at any stage of life,” said Billy Dunn,

M.D., director of the Division of Neurology Products in the FDA’s Center

for Drug Evaluation and Research. “As shown by our suggestion to the

sponsor to analyze the results of the study earlier than planned, the

FDA is committed to assisting with the development and approval of safe

and effective drugs for rare diseases and we worked hard to review this

application quickly; we could not be more pleased to have the first

approved treatment for this debilitating disease.”</p><p>SMA is a

hereditary disease that causes weakness and muscle wasting because of

the loss of lower motor neurons controlling movement. There is wide

variability in age of onset, symptoms and rate of progression. Spinraza

is approved for use across the range of spinal muscular atrophy

patients.</p>