Biogen Inc announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending a marketing authorization under exceptional circumstances for QALSODY® (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene. If authorized by the European Commission (EC), QALSODY will be the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).
“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,” said Priya Singhal, M.D., M.P.H., Head of Development at Biogen. “We are proud to help pioneer the role of neurofilament in SOD1-ALS clinical trials and are deeply grateful to the people living with SOD1-ALS, their loved ones and study care teams for their dedication to furthering research for the ALS community.”
The CHMP’s recommendation for QALSODY is based on the totality of evidence, including the targeted mechanism of action, biomarker and clinical data. In the 28-week Phase 3 VALOR study, reductions of 60% in plasma neurofilament light chain (NfL) were observed in participants who received QALSODY compared to the placebo group, suggesting reduced neuronal injury. Trends towards improvement in the physical abilities of participants who received QALSODY were seen compared to those who received placebo, as measured by the ALS Functional Ratings Scale-Revised (ALSFRS-R). The most common side effects that occurred in ≥10% of QALSODY treated participants and more than the placebo arm were pain, fatigue, fever, joint pain, muscle pain and increased levels of white blood cells and proteins in the cerebrospinal fluid. Serious neurologic events, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis have also been reported.
“The CHMP’s recommendation in support of QALSODY approval provides new hope for the ALS community in Europe,” said Philip Van Damme, M.D., Ph.D., professor of neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium. “This is a significant milestone for the entire ALS community - for the first time we have a treatment that led to sustained reductions in neurofilament, a marker of axonal injury and neurodegeneration. The QALSODY development program has provided critical learnings on clinical trial design and the use of biomarkers that is advancing the entire field.”
A marketing authorization under exceptional circumstances is recommended when the benefit/risk assessment is determined to be positive but due to the rarity of the disease, it is unlikely that comprehensive data can be obtained under normal conditions of use. 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.
QALSODY® (tofersen) is an antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA to reduce SOD1 protein production. The U.S. Food and Drug Administration granted accelerated approval for QALSODY to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).
