The U.S. Food & Drug Administration (FDA) has approved Biogen Inc.’s nusinersen drug for treating children and adults suffering form the spinal muscular atrophy (SMA) genetic disease.

Nusinersen is an antisense oligonucleotide that boosts concentrations of survival motor neuron (SMN) protein, which gets under-expressed when a patient is hit by SMA. The drug is administered with the help of injection into the patient’s cerebrospinal fluid.

A clinical trial involving 121 patients with infantile-onset SMA showed the efficacy of the Biogen drug. The participants were randomly allocated to receive nusinersen or a mock procedure in control group.

Results showed that 40 per cent of patients who received nusinersen gained improvement in the motor milestones, while none of the patients in the control group showed improvement.

The FDA sais in a statement, “The findings appeared generally supportive of the clinical efficacy demonstrated in the controlled clinical trial in infantile-onset patients … there has been a long-standing need for a treatment for [SMA], the most common genetic cause of death in infants, and a disease that can affect people at any stage of life.”

SMA is a rare and often fatal genetic disease that affects nearly 1 in every 11,000 newborns. This progressive motor neuron disorder causes symptoms like muscle weakness and difficulties in eating and breathing.