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

 
• By Mandy Jackson

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.

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.

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