Qalsody(tofersen)

Qalsody (tofersen) is indicated for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. It received an orphan medicinal product designation on 29 August 2016, and a marketing authorization application was submitted on 31 October 2022. A CHMP positive opinion was issued on 22 February 2024, with a revised opinion on 19 April 2024. The marketing authorization was recommended under exceptional circumstances.

In SOD1-G93A mice, tofersen demonstrated efficacy in delaying disease onset and extending survival. Median disease onset was 168 days with tofersen compared to 147.5 days in control ASO mice at 30 days of age. Median survival was 193.5 days with tofersen versus 170 days in control ASO mice at 30 days of age. Tofersen also showed a median reduction in CSF SOD1 of 23% and a median reduction in plasma NFL of 39% based on PKPD modeling.

Safety findings for tofersen 100 mg (Pool RC1) included a fall incidence of 23.6% and back pain incidence of 19.4%. Participants with events related to lumbar puncture were 80.6% for tofersen 100 mg (Pool RC1) and 80.6% for placebo (Pool RC2). Treatment emergent ADAs were observed in 58.4% of participants. The recommended dose of tofersen is 100 mg.