Biogen/Ionis’s Spinraza Approved; Second Antisense Drug For Neurodegeneration In 2016

Analysts expect strong uptake for the antisense oligonucleotide (ASO) given the high unmet need in SMA and positive data in multiple studies, while the first ASO approved this year – Sarepta’s Exondys 51 – has seen some pushback from payers despite the lack of DMD therapies.

FDA approved background, 3D rendering, blue street sign

Biogen and partner Ionis Pharmaceuticals Inc. are ending 2016 with US FDA approval for Spinraza (nusinersen) in the treatment of spinal muscular atrophy (SMA), marking the second antisense drug approved this year to treat a rare neurodegenerative disease.

Spinraza follows in the footsteps of the controversial approval of Sarepta Therapeutics Inc.’s Exondys 51 (eteplirsen) for Duchenne muscular...

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