A pedestrian walks past Biogen Inc. headquarters in Cambridge, Massachusetts, on Monday, June 7, 2021.
Adam Glanzman | Bloomberg | Getty Images
The Food and Drug Administration on Tuesday granted accelerated approval for Biogen‘s drug tofersen, which treats a rare and aggressive form of the disease known as ALS.
Accelerated approval is an FDA designation that clears drugs faster if they fill an unmet medical need for serious conditions. The approval requires Biogen and its co-developer Ionis to further study tofersen and verify its clinical benefits.
If a subsequent trial confirms those benefits, the FDA can grant traditional approval for the drug.
The FDA said its decision is based on mixed late-stage trial results published in 2021, which indicated that tofersen significantly reduced a key protein called neurofilament light. That protein is associated with the severity of the mind-wasting disease.
“The findings are reasonably likely to predict a clinical benefit in patients,” the agency said in a press release.
An independent panel of advisors to the FDA last month similarly voted that tofersen’s effect on neurofilament could produce a clinical benefit in ALS patients.
Biogen CEO Chris Viehbacher said in a statement the FDA’s decision is a “pivotal moment in ALS research.”
“We gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS,” Viehbacher said. “We believe this important scientific advancement will further accelerate innovative drug development for ALS.”
ALS, commonly known as Lou Gehrig’s disease, is a progressive and fatal neuromuscular disease that causes nerve cells in the brain and spinal cord to break down over time.
Tofersen specifically targets a form of ALS in people with mutations in a specific gene, which are passed down through generations within families.
Those mutations can cause a protein called SOD1 to accumulate to toxic levels, which damages the…
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