FDA Extends Review of Tofersen


Biogen Inc. announced that the U.S. Food and Drug Administration extended the review period of the promising gene therapy treatment Tofersen to consider additional data. The initial review period, which was announced in July 2022, was scheduled to conclude in January 2023. The review will now be extended to April 25, 2023.

Extensions of the review period under the Prescription Drug User Fee Act are not uncommon, particularly when new data is presented to the agency as part of its consideration of the safety and efficacy of a treatment under review. We are encouraged that the agency is considering additional data and thank Biogen for its commitment to maintaining its early access program throughout the extended review.

Nevertheless, we recognize that this is disappointing news for those hoping for potential approval. Though an additional three-month wait may not seem significant, for those living with ALS and their families this news is likely devastating. As such, we strongly encourage the FDA to consider Tofersen as soon as possible and to act with urgency to approve all new ALS treatments that are safe and effective. People living with ALS cannot wait and the FDA should do everything it can to ensure that they do not have to.

Click here to learn more about Tofersen.