Spinraza by Biogen is a medicine used to treat 5q spinal muscular atrophy (SMA), a genetic disease that causes weakness and wasting of the muscles including the lung muscles. The disease is linked to a defect on chromosome 5q and symptoms usually start shortly after birth.
Because the number of patients with SMA is low, the disease is considered ‘rare’, and Spinraza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 2 April 2012.
Spinraza contains the active substance nusinersen.
Was it successful? It is the first question comes to mind before buying the most expensive medicine of the century.
But answer is yes. One main study, involving 121 babies (of an average age of 7 months) with SMA, showed that Spinraza is effective in improving movement when compared to placebo (sham injection).
After one year of treatment, 51% of babies receiving Spinraza (37 out of 73) showed progress in developing head control, rolling, sitting, crawling, standing and walking, whereas no similar progress was seen in any of the babies who received placebo. In addition, most babies treated with Spinraza survived for longer and needed breathing support later than those given placebo.
Another study assessed Spinraza’s effectiveness in children whose SMA was less severe and diagnosed at a later stage (average age of 3 years). After 15 months of treatment, 57% of children receiving Spinraza showed improvement in movement compared with 26% of children on placebo.