Ionis Announces European Medicines Agency Accepts Marketing Authorization Application of Tofersen to Treat Rare, Genetic form of ALS

Ionis Pharmaceuticals has 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

The EMA is the second regulatory agency to accept review of a marketing application for tofersen following U.S. Food and Drug Administration (FDA) acceptance earlier this year. The FDA has set a PDUFA date for tofersen of April 25, 2023. There are currently no treatments targeted for SOD1-ALS.

“EMA acceptance of the tofersen MAA is a significant development for people and families battling SOD1-ALS,” said C. Frank Bennett, Ph.D., executive vice president, chief scientific officer and franchise leader for neurological programs at Ionis. “If approved, tofersen will be the world’s first treatment that targets a genetic cause of ALS. Tofersen also strengthens Ionis’ platform strategy to target other neurological diseases, including other forms of ALS.”

The MAA includes results from the Phase 3 VALOR study, its open-label extension (OLE) study, a Phase 1 study in healthy volunteers and a Phase 1/2 study evaluating ascending dose levels. Also included are the most current 12-month integrated results from VALOR and the OLE study that were recently published in The New England Journal of Medicine. Biogen announced that it will maintain its early access program for tofersen, which is now available in 34 countries. Biogen also announced that it will continue to actively engage with other regulators around the world and will provide updates when appropriate.