Biogen announces EMA accepted tofersen MAA to treat rare form of ALS

Biogen announced the European Medicines Agency, EMA, has accepted the Marketing Authorization Application, MAA, for review of tofersen, an investigational drug for the treatment of superoxide dismutase 1, SOD1, amyotrophic lateral sclerosis, ALS. SOD1-ALS is a progressive and uniformly fatal disease that affects less than 1,000 people in Europe. There is currently no treatment targeted for SOD1-ALS. "Through our clinical development program, we have seen that tofersen has the potential to slow the progression of this relentless and ultimately fatal disease," said Priya Singhal, M.D., M.P.H., Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen. "Regulatory submissions in the U.S. and now EU represent an important step in our efforts to bring the first genetically-targeted treatment for SOD1-ALS to the ALS community as quickly as possible." The MAA 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. 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. Tofersen is also under review with the U.S. Food and Drug Administration with Priority Review and has a Prescription Drug User Fee Act action date of April 25, 2023.