2024-02-26 10:09:39
Biogen today announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion regarding the marketing authorization under exceptional circumstances for QALSODY® (tofersen) in the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. If the European Commission grants Marketing Authorization, QALSODY will become the first treatment targeting a genetic cause of ALS, a motor neurone disease, to be authorized in the European Union.
“The positive CHMP opinion highlights the importance of QALSODY for the treatment of ALS-SOD1 and further demonstrates Biogen’s commitment to addressing the unmet needs of people with ALS and other neuromuscular diseases said Priya Singhal, MD and Global Chief Development Officer of Biogen. “We are proud to help highlight the role of neurofilament in SOD1-ALS clinical trials and are deeply grateful to those who participated in the studies – people with SOD1-ALS and their loved ones as well as those who participated in the studies. caregivers – for their dedication to advancing ALS research. »
The CHMP recommendation for QALSODY is based on the full body of evidence, including the targeted mechanism of action, biomarkers and clinical data. During week 28 of Phase 3 of the VALOR study, a 60% reduction in plasma neurofilament light chain (NfL) was observed in participants who received QALSODY compared to the placebo group, suggesting a reduction of neuronal damage. Trends toward improvement in physical abilities of participants who received QALSODY were observed compared to those who received placebo, as measured by the ALS Functional Ratings Scale-Revised (ALSFRS-R). The most common adverse reactions reported in patients treated with tofersen were: pain, arthralgia, fatigue, increased CSF white blood cells, increased protein, myalgia and fever.
“The CHMP recommendation for marketing authorization of QALSODY brings hope to the ALS community in Europe,” said Philip Van Damme, MD, Ph.D., Professor of neurology and director of the Neuromuscular Reference Center at the University Hospital of Louvain in Belgium. “This is an important milestone for the entire ALS community – for the first time, we have a treatment that has resulted in lasting reductions in neurofilaments, a marker of axonal damage and neurodegeneration. » QALSODY’s clinical development program has provided essential knowledge in clinical trial design and the use of biomarkers, enabling advancements in this area. »
The CHMP may recommend marketing authorization in exceptional circumstances where the benefit/risk assessment is judged to be positive but, due to the rarity of the disease, it is unlikely that complete data can be obtained under normal conditions of use. The CHMP recommendation for QALSODY will now be considered by the European Commission for a marketing authorization decision in the European Union, with the final decision expected in the second quarter of 2024.
References :
1. Brown CA, Lally C, Kupelian V, Flanders WD. Estimated Prevalence and Incidence of Amyotrophic Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants. Neuroepidemiology. 2021;55(5):342-353. doi : 10.1159/000516752. Publication électronique, 9 juillet 2021
2. QALSODY Prescribing Information, Cambridge, MA : Biogen.
3. National Institute of Neurological Disorders and Stroke. Amyotrophic Lateral Sclerosis (ALS). Available at: https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als. Last access: April 2023
4. Akcimen F, Lopez ER, Landers JE, et al. Amyotrophic lateral sclerosis: translating genetic discoveries into therapies. Nat Rev Genet. 2023. https://doi.org/10.1038/s41576-023-00592-y
Source and visual: Biogen
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