BRIEF—EMA accepts Biogen's tofersen for review

The European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for review of US biotech major Biogen’s 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.

As there is currently no treatment targeted for SOD1-ALS, if approved, tofersen would be the first for this indication.

“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 Dr Priya Singhal, head of global safety and regulatory sciences and interim head of R&D at Biogen, adding: “Regulatory submissions in the US 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.”

Tofersen is also under review with the US Food and Drug Administration with Priority Review and has a Prescription Drug User Fee Act action date of April 25, 2023.

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

Biogen continues to actively engage with other regulators around the world and will provide updates when appropriate.