FDA pushes back decision date for Biogen NDA

Oct. 17, 2022

Massachusetts-based Biogen announced this week that the FDA has extended its review period for the new drug application of its ALS treatment, tofersen. 

The new Prescription Drug User Fee Act (PDUFA) goal date is April 25, 2023 — 3 months later than anticipated — which will allow the agency to review information requests considered major amendments.

Tofersen is an investigational superoxide dismutase 1 (SOD1) treatment for amyotrophic lateral sclerosis. Also known as BIIB067, the drug is an antisense oligonucleotide and works by blocking the production of the SOD1 protein, which clumps together with copies of itself and damages the nervous system.

Biogen initially licensed the drug candidate from RNA-biotech Ionis Pharmaceuticals. In June of this year, the two shared phase 3 trial results showing that it led to robust and sustained reductions in neurofilament, a marker of neurodegeneration. 

Just a few weeks ago, Amylyx finally scored FDA approval for its ALS drug, now branded as Relyvrio, adding another important treatment option for the life-threatening disease that currently has no cure.