CAMBRIDGE, Mass. - Biogen Inc. (NASDAQ:BIIB) announced a significant development in the treatment of amyotrophic lateral sclerosis (ALS), as the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization for its drug QALSODY® (tofersen). This recommendation is specifically for the treatment of adult patients with ALS linked to a mutation in the superoxide dismutase 1 (SOD1) gene.
The CHMP's positive opinion is a step towards QALSODY becoming the first therapy in the European Union targeting a genetic cause of ALS, also known as motor neuron disease (MND). The recommendation is based on the totality of evidence from clinical trials, including the Phase 3 VALOR study, which demonstrated a 60% reduction in plasma neurofilament light chain (NfL) in treated patients, indicating reduced neuronal injury.
QALSODY's development has been noted for its role in pioneering the use of neurofilament as a biomarker in clinical trials for ALS. The drug, an antisense oligonucleotide (ASO) designed to reduce SOD1 protein production, has already been granted accelerated approval in the U.S. for the same indication.
The most common side effects reported in QALSODY-treated participants were pain, fatigue, fever, joint pain, muscle pain, and increased levels of white blood cells and proteins in the cerebrospinal fluid. Serious neurologic events such as myelitis, radiculitis, papilledema, elevated intracranial pressure, and aseptic meningitis have also been reported.
A marketing authorization under exceptional circumstances is considered when comprehensive data is unlikely due to the rarity of the disease, but the benefit/risk assessment is positive. The European Commission is expected to make a final decision on the marketing authorization for QALSODY in the European Union in the second quarter of 2024.
This news follows Biogen's continuous efforts in ALS research and drug development, despite setbacks in the past. The company's commitment to addressing the unmet needs of those living with ALS and neuromuscular diseases is reinforced by this recent advancement.
Investors and the medical community will be watching closely as the European Commission reviews the CHMP's recommendation, which could introduce a new treatment option for the small population affected by SOD1-ALS in Europe. The information in this article is based on a press release statement from Biogen Inc.
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