European Medicines Agency Accepts Tofersen Marketing Authorization Application to Treat Rare, Genetic Form of ALS

European Medicines Agency Accepts Tofersen Marketing Authorization Application to Treat Rare, Genetic Form of ALS

 Biogen Inc. (Nasdaq: BIIB) 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.2 There is currently no treatment targeted for SOD1-ALS.3

“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 (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.

“The effects we have seen with tofersen diverge from the natural history of the disease and bring hope for the treatment of SOD1-ALS,” said Philip Van Damme, M.D., Ph.D., professor of neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium. “Today’s announcement is an important milestone for the ALS community in Europe where there is a tremendous need for additional treatment options.”

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.

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.

About Tofersen
Tofersen is an antisense drug being evaluated as a treatment of SOD1-ALS. Tofersen is designed to bind to SOD1 mRNA, inducing its degradation by RNase-H to reduce SOD1 protein production. In addition to the ongoing open label extension of VALOR, tofersen is being studied in the Phase 3 ATLAS study designed to evaluate whether tofersen can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement.

About Amyotrophic Lateral Sclerosis and SOD1-ALS
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.3

Multiple genes have been implicated in ALS. Genetic testing helps determine if a person’s ALS is associated with a genetic mutation, even in individuals without a family history of the disease. Currently, there are no genetically targeted treatment options for ALS. Mutations in the SOD1 gene are responsible for approximately 2 percent of the estimated 168,000 people who have ALS globally (SOD1-ALS).2 Life expectancy in SOD1-ALS varies widely with some patients surviving less than a year.4

Biogen’s Continuous Commitment to ALS
For over a decade, Biogen has been committed to advancing ALS research to provide a deeper understanding of all forms of the disease. The company has continued to invest in and pioneer research despite making the difficult decision to discontinue a late-stage ALS asset in 2013. Biogen has applied important learnings to its portfolio of assets for genetic and other forms of ALS, with the goal of increasing the probability of bringing a potential therapy to patients in need. These applied learnings include evaluating genetically validated targets in defined patient populations, pursuing the most appropriate modality for each target and employing sensitive clinical endpoints. Today, the company has a pipeline of investigational drugs being evaluated in ALS, including tofersen and BIIB105.

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