Biogen's Qalsody receives positive opinion from CHMP for ALS treatment in Europe
As a result, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Qalsody (tofersen).
This recommendation is for the treatment of adults with ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene. If approved by the European Commission (EC), Qalsody will mark a groundbreaking milestone as the first treatment targeting a genetic cause of ALS in the European Union.
Dr Priya Singhal, head of development at Biogen, expressed the company's commitment to addressing the unmet needs of ALS patients and neuromuscular diseases.
She said: “The CHMP’s positive opinion reinforces the impact Qalsody can have in SOD1-ALS and further demonstrates Biogen’s commitment to address the unmet needs of people living with ALS and neuromuscular diseases.”
Improvement in ALS physical abilities
The recommendation for Qalsody is based on a comprehensive evaluation of evidence, including its targeted mechanism of action, biomarker, and clinical data.
The phase 3 Valor study, conducted over 28 weeks, demonstrated promising results, with participants receiving Qalsody showing a 60% reduction in plasma neurofilament light chain (NfL) compared to the placebo group.
Additionally, trends towards improvement in physical abilities were observed in Qalsody-treated participants compared to those receiving placebo, as measured by the ALS Functional Ratings Scale-Revised (ALSFRS-R).
Dr Philip Van Damme, professor of neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium, highlighted the significance of Qalsody's development program in providing critical insights into clinical trial design and the use of biomarkers.
Tofersen - an antisense oligonucleotide
He said: “For the first time, we have a treatment that led to sustained reductions in neurofilament, a marker of axonal injury and neurodegeneration.”
A marketing authorization under exceptional circumstances is recommended when the benefit/risk assessment is positive, but comprehensive data cannot be obtained due to the rarity of the disease. CHMP's recommendation for Qalsody will undergo review by the EC, with a decision expected in the second quarter of 2024.
Tofersen is an antisense oligonucleotide (ASO) designed to reduce SOD1 protein production by binding to SOD1 mRNA. The U.S. Food and Drug Administration (FDA) has granted accelerated approval for Qalsody to treat ALS in adults with a mutation in the SOD1 gene, based on observed reductions in plasma neurofilament light chain (NfL). Biogen licensed Qalsody from Ionis Pharmaceuticals, Inc., and it was discovered by Ionis.
In addition to the ongoing Phase 3 Valor study's open-label extension, Qalsody is being evaluated in the phase 3 ATLAS study, aiming to delay clinical onset in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity.
