Ionis Pharmaceuticals announced that the European Medicines Agency has accepted the marketing authorization application for review of tofersen, an investigational medicine for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis. SOD1-ALS is a progressive and uniformly fatal disease that affects fewer than 1,000 people across Europe. The FDA has set a PDUFA date for tofersen of April 25, 2023. There are currently no treatments targeted for SOD1-ALS.
Published first on TheFly
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