Biogen's Tofersen MAA Accepted In Europe To Treat Rare, Genetic Form Of ALS

(RTTNews) - Biogen Inc. (BIIB) announced Monday that the European Medicines Agency or EMA has accepted the Marketing Authorization Application or MAA for review of tofersen to treat rare, genetic form of amyotrophic lateral sclerosis or ALS.

Tofersen is an investigational drug for the treatment of superoxide dismutase 1 or SOD1- ALS, a progressive and uniformly fatal disease. SOD1-ALS is a rare, genetic form of ALS, comprising around 2% of people with ALS, and there is currently no treatment.

If approved, tofersen, an antisense drug, would be the first treatment to target the genetic cause of ALS.

The MAA includes results from the Phase 3 VALOR study, its open label extension or 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.

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.

Priya Singhal, Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen, said, "Through our clinical development program, we have seen that tofersen has the potential to slow the progression of this relentless and ultimately fatal disease. 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."

Biogen said it will maintain its early access program for tofersen, which is now available in 34 countries.

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