Ionis Announces EMA's Acceptance Of Marketing Authorization Application Of Tofersen For SOD1-ALS

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) Monday announced that the European Medicines Agency or EMA has accepted the marketing authorization application for its tofersen for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis or SOD1-ALS.

Earlier FDA had accepted for review of a marketing application and has set a PDUFA date for tofersen of April 25, 2023

Superoxide dismutase 1 or SOD1-ALS is a progressive and uniformly fatal disease that affects fewer than 1,000 people across Europe. There are currently no treatments targeted for SOD1-ALS.

The marketing authorization application includes results from the Phase 3 VALOR study, its open-label extension study, a Phase 1 study in healthy volunteers and a Phase 1/2 study evaluating ascending dose levels.