Authorizing the nation’s first injectable drug targeting the rare, lethal disease of spinal muscular atrophy (SMA), the U.S. Food and Drug Administration recently approved Spinraza (nusinersen) as a treatment for both children and adults.
SMA is a genetic ailment impacting muscular strength and movement. Age on onset, symptoms and progression vary among individuals, but all victims suffer weakness and muscle wasting.
Approved for the entire range of SMA patients, Spinraza is administered by injection into the spinal cord fluid. The drug was approved following clinical studies in which the FDA collaborated with a sponsor, and subsequently was expedited for quick review.
During the clinical trials — involving 121 patients with early onset and diagnosis — two-thirds of the group was injected with the drug while one-third received a mock procedure with no drug injected. Researchers then compared the proportion of patients who experienced motor improvements including head control, sitting, ability to kick in supine position, rolling, crawling, standing and walking.
Results revealed that 40 percent of those injected with Spinraza improved, whereas none of the control participants did. The treatment has been granted “orphan drug” status, meaning that it will be given priority as a treatment for rare diseases.
Spinraza was developed by Ionis Pharmaceuticals of Carlsbad, California and is marketed by Cambridge, Massachusetts-based Biogen.
