FDA Accelerated Approval of Tofersen Highlights Importance of Blood Neurofilament Light Chain as Surrogate Endpoint in Neurology Therapeutic Trials
Blood-based measurement of neurofilament light chain (NfL) provides compelling evidence to support an accelerated drug approval by the FDA. The decision has positive implications for NfL blood testing as a key tool in neuro-drug development.
Tofersen is designed to reduce the overproduction of SOD1 protein in SOD1-ALS patients. In the Phase III VALOR trial, the drug did not significantly slow patients’ clinical decline. However, there were positive trends in the data, and open label extension results have since shown evidence of clinical benefits along with highly significant reductions of secondary endpoint biomarkers in favor of a positive treatment effect. This data supported an extended FDA review of tofersen’s new drug application under the accelerated pathway leading to its recent announcement of approval. Critical to the approval were plasma NfL trends, with the
The FDA’s decision to approve tofersen through its Accelerated Approval Program focused on strong surrogate biomarker data, adding momentum to the use of biomarkers in predicting disease severity and clinical benefit in neurodegenerative diseases. NfL, in particular, has been extensively studied in different neurodegenerative diseases, including Alzheimer’s, spinal muscular atrophy, hereditary transthyretin-mediated amyloidosis, and multiple sclerosis. In ALS, independent studies have shown NfL levels correlate with disease severity, disease progression rate, and survival. Alternatively, therapy-mediated reductions in NfL have been correlated with clinical improvement from approved drugs for other neurological diseases, supporting the use of NfL as a critical pharmacodynamic marker.
“An important advance stemming from the tofersen approval is a demonstration of the potential of NfL as a potential surrogate biomarker that can serve as a leading indicator of drug efficacy for some investigational therapies in neurodegenerative disorders,” said
“Blood-based NfL measurements are materially advancing and accelerating therapeutics development for, and clinical management of, patients with neurodegenerative diseases, including Alzheimer’s, spinal muscular atrophy, hereditary transthyretin-mediated amyloidosis, and multiple sclerosis,” said
To learn more about Quanterix’s Simoa® NfL assay, visit: https://quanterix.com/products-and-services/simoa-neurofilament-light-nfl-ldt/
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For more information about Quanterix’s work in neurology, visit: https://www.quanterix.com/therapeutic-areas/neurology/.
From discovery to diagnostics, Quanterix’s ultrasensitive biomarker detection is fueling breakthroughs only made possible through its unparalleled sensitivity and flexibility. The Company’s Simoa® technology has delivered the gold standard for earlier biomarker detection in blood, serum or plasma, with the ability to quantify proteins that are far lower than the Limit of Quantification (LoQ) of conventional analog methods. Its industry-leading precision instruments, digital immunoassay technology and CLIA-certified Accelerator laboratory have supported research that advances disease understanding and management in neurology, oncology, immunology, cardiology and infectious disease.
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