FDA grants Biogen ALS drug accelerated approval

April 26, 2023

The U.S. FDA has approved Biogen's Qalsody for the treatment of an ultra rare form of amyotrophic lateral sclerosis (ALS).

The drug was approved for the small subset of ALS patients who have a mutation in the superoxide dismutase 1 (SOD1) gene. According to Biogen, the uniformly fatal mutation affects approximately 330 people in the U.S. 

Qalsody, which was developed in collaboration with Ionis Pharmaceuticals, is an antisense oligonucleotide that works by blocking the production of the SOD1 protein, which clumps together with copies of itself and damages the nervous system. It is now the first approved treatment to target a genetic cause of ALS.

The treatment was given the green light by the FDA under accelerated approval — meaning continued approval hinges on verification of clinical benefit in confirmatory trials. Biogen's ongoing phase 3 ATLAS study in people with presymptomatic SOD1-ALS will serve as the confirmatory trial.

The decision follows the recommendation of the FDA's Peripheral and Central Nervous System Drugs Advisory Committee who back in March voted unanimously for consideration of a potential accelerated approval. 

This is the second ALS approval in the past year. Back in September Amylyx scored FDA approval for its broader ALS drug, now branded as Relyvrio. Relyvrio is an oral fixed-dose combination of sodium phenylbutyrate and taurursodiol, designed to preserve neurons by blocking cell death pathways in the mitochondria and endoplasmic reticulum.