Tofersen: First Approval

Tofersen (Qalsody((TM))) is an antisense oligonucleotide being developed by Biogen for the treatment of amyotrophic lateral sclerosis (ALS). On 25 April 2023, tofersen was approved in the USA for the treatment of ALS in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This article summarizes the milestones in the development of tofersen leading to this first approval for ALS.

Automated summary

Tofersen (Qalsody(TM)) is an antisense oligonucleotide developed by Biogen to treat amyotrophic lateral sclerosis (ALS). It was approved in the USA on April 25, 2023, for the treatment of ALS in adults with a mutation in the superoxide dismutase 1 (SOD1) gene. This article summarizes the developmental milestones of tofersen leading to its first approval for ALS.