Biogen/Ionis’ Spinraza Data Shows Opportunity For Broad SMA Label

Both companies’ stocks surged Nov. 7 on positive data from a second Phase III trial showing the antisense drug improved motor function in children with later onset spinal muscular atrophy.

With positive data from a second Phase III trial testing Biogen Inc./Ionis Pharmaceuticals Inc.’s Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) now in hand, the antisense drug appears on track to be the first drug approved by FDA for the genetic rare disease, and poised for a broad label.

Biogen is anticipating a rapid approval and said it is preparing to launch Spinraza by the end of the year...

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