Ionis announces European Medicines Agency accepts Marketing Authorization Application of tofersen to treat rare, genetic form of ALS

SOD1-ALS affects approximately 2% of people living with ALS worldwide1 If approved, tofersen would be the world's first treatment to target a genetic cause of ALS EMA acceptance follows FDA's acceptance of tofersen NDA earlier this year CARLSBAD, Calif., Dec. 5, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for review of tofersen, an investigational medicine for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). SOD1-ALS is a progressive and uniformly fatal disease that affects fewer than 1,000 people across Europe.2

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