Biogen Wins Accelerated Approval for ALS Drug

The FDA has granted Biogen conditional approval for antisense oligonucleotide Qalsody (tofersen) for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the SOD1 (superoxide dismutase 1) gene, producing a rare, hereditary form of the disease. Qalsody is the first approved treatment to target a genetic cause of ALS. Tofersen was discovered by Ionis Pharmaceuticals. Biogen licensed tofersen from Ionis under a collaborative development and license agreement in 2018.

The accelerated approval is based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with Qalsody. Neurofilaments are proteins that are released from neurons when they are damaged, making them a marker of neurodegeneration. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

“For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS,” said Christopher A. Viehbacher, President and Chief Executive Officer of Biogen. “Today also marks a pivotal moment in ALS research as 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. We believe this important scientific advancement will further accelerate innovative drug development for ALS.”

Qalsody is also being studied in a Phase 3 study to evaluate whether it can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity (elevated plasma NfL). (Source: Biogen Website, 25 April, 2023)