NETHERLANDS —The European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for review of tofersen to treat rare, genetic form of amyotrophic lateral sclerosis (ALS) being developed by US biotech major Biogen.
Tofersen is an investigational drug for the treatment of superoxide dismutase 1 (SOD1)- ALS, a progressive and uniformly fatal disease.
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.
SOD1-ALS comprises 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 antisense oligonucleotide drug tofersen has been designed to bind to SOD1 mRNA, prompting its degradation and reducing SOD1 protein production.
Tofersen also strengthens Ionis’ platform strategy to target other neurological diseases, including other forms of ALS.
The EMA is the second regulatory agency to accept review of a marketing application for tofersen following US Food and Drug Administration (FDA) acceptance earlier this year.
According to the release, tofersen is also under review by the FDA after the agency accepted a new drug application for the treatment in July. A decision from the FDA is due in April 2023.
“EMA acceptance of the tofersen MAA is a significant development for people and families battling SOD1-ALS,” said C. Frank Bennett, Ph.D., executive vice president, chief scientific officer and franchise leader for neurological programs at Ionis. “
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. Patients with some SOD1 mutations have an even shorter life expectancy.
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.
Biogen announced that it will maintain its early access program for tofersen, which is now available in 34 countries.
Biogen also announced that it will continue to actively engage with other regulators around the world and will provide updates when appropriate.