A new study showed spinal muscular atrophy (SMA) patients functionally improved while taking the drug nusinersen, also known as Spinraza.
A news announcement from Ionis Pharmaceuticals and Biogen said children with type 2 SMA, sometimes referred to as later-onset SMA, “experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment.”
The companies added that the drug “demonstrated a favorable safety profile in the study.”
Ionis Pharmaceuticals and Biogen are working together on the development and launch of Spinraza. The drug was also been found to benefit children with infantile-onset SMA (stage 1) in a different study.
“These results, along with our successful trial in infantile-onset SMA, reinforce the potential of Spinraza to benefit a broad range of SMA patients,” said Michael Ehlers, M.D., Ph.D., Biogen’s executive VP and head of research & development. “We will make regulators around the globe aware of this data and will continue working closely with them to bring Spinraza to families affected by SMA as quickly as possible.”
SMA currently has no treatment or cure. It is the number one genetic cause of death for infants worldwide.
The SMA type 2 study, called CHERISH, included 126 children with type 2 SMA. The children ranged in age from 2 to 12 years old.
Following the positive results, the CHERISH study was halted, and children participating in it — those who were taking Spinraza and those who did not receive the drug — can enroll in an open-label study that will provide Spinraza to all its participants. The open-label study, named SHINE, will examine the long-term effects and tolerability of Spinraza.
The drug has received special status from the U.S. Food & Drug Administration designed to expedite its availability. Biogen said it hopes to begin distributing Spinraza this year or in early 2017.
