Tofersen receives positive opinion from CHMP (EMA)

The Committe of Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending a marketing authorization for QALSODY (better known as Tofersen). If authorized by the European Commission (EC), QALSODY (Tofersen) will be approved in the European Union as a treatment for people with ALS, with the SOD1 mutation. 

Valor study 

The recommendation is based on the results of the VALOR study, as presented on 22 september 2022. These results showed that for participants with the SOD1 mutation, who received the drug for a longer period of time, the amount of SOD1 protein was reduced. After 28 weeks, the amount of neurofilaments (a marker of neuronal injury) was reduced by 60%. Trends towards improvements in physical abilities of participants who received QALSODY compared to placebo were also observed.  

Treatment and use of Tofersen 

QALSODY/Tofersen is an antisense oligonucleotide designed to bind to SOD1 messenger RNA, marking it for destruction by cellular systems, thereby reducing the amount of toxic protein present. Patients receive the drug by lumbar puncture monthly.  

The CHMP’s recommendation for QALSODY will now be reviewed by the EC for a decision on a marketing authorization in the European Union, with a decision expected in the second quarter of 2024. 

Reaction TRICALS 

“We are incredibly happy to hear about the positive advice of the EMA regarding the marketing authorization of QALSODY/Tofersen. This news is a big milestone for the ALS field. We want to thank all participants of the VALOR study, and all study care teams for their time and effort in making this trial a success.” Prof. Leonard H. van den Berg, Chair of TRICALS, Professor of Neurology at the University Medical Centre Utrecht, The Netherlands. 

More information?

Read the summary of the positive opinion for Qalsody of the EMA here

Read the official statement of Biogen here