If approved, tofersen would become the first treatment in Europe to target a genetic cause of ALS.
Tofersen is already under review with the US Food and Drug Administration (FDA), with an expected decision date of April 25 (no timescales have been given for the EMA application).
Phase 3 data
The Marketing Authorization Application in Europe includes results from the Phase 3 Valor study and the open-label-extension 12 month data (published in August, this additional data showed a slowed decline in clinical function, respiratory function, strength, and quality of life with early initiation of tofersen).
Amyotrophic lateral sclerosis (ALS) is a rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement. People with ALS experience muscle weakness and atrophy, causing them to lose independence as they steadily lose the ability to move, speak, eat, and eventually breathe. Average life expectancy for people with ALS is three to five years from time of symptom onset.
Mutations in the SOD1 gene are responsible for around 2% of the estimated 168,000 people who have ALS globally, leading to the production of a toxic form of the SOD1 protein. In Europe, the disease affects around 1,000 people.
Known as gene silencing, tofersen binds and degrades SOD1 mRNA to reduce synthesis of SOD1 protein production.
“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 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.”