FDA Pushes Back Action Date for Biogen’s Tofersen NDA for ALS

The FDA has pushed back its decision date on Biogen’s New Drug Application for tofersen for the treatment of patients with superoxide dismutase type 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare genetic form of ALS, by three months from Jan. 25, 2023 to April 25, 2023.

The FDA is requiring the additional review time after the company’s responses to the agency’s information requests led to a major amendment to the company’s application.

Tofersen uses antisense oligonucleotides to target messenger RNA (mRNA) to reduce SOD1 protein production. Superoxide is by-product of oxygen metabolism that can cause cell damage.

While the FDA review is ongoing, the company will continue to maintain the early access program for tofersen.