Blood-based biomarker test can be used to assess disease activity in patients with multiple sclerosis and for treatment response monitoring.
The FDA has granted Breakthrough Device designation for a blood-based test to assess the risk of disease activity in patients with relapsing-remitting multiple sclerosis (RRMS).
FDA granted the designation to Quanterix Corp. for its Simoa neurofilament light chain (NfL) plasma test, a digital immunoassay that measures NfL in human serum and plasma. The test can be used in conjunction with clinical, imaging and laboratory findings to help identify multiple sclerosis patients who are at lower or higher risk for relapse within four years, Quanterix said in a statement.
“For the more than 2 million people suffering from MS worldwide, there’s an important need for more informed and effective treatment options,” said Kevin Hrusovsky, executive chairman of the board of Quanterix and founder of Powering Precision Health, a nonprofit organization that convenes an annual summit of biomarker researchers.
The Simoa NfL test was the first such test to be available to measure NfL in blood, Quanterix CEO Masoud Toloue, Ph.D., told Managed Healthcare Executive®. It has been available for research purposes since 2017.
“The measurement of NfL in blood (serum/plasma) was a breakthrough because previously the protein could only be measured in the cerebrospinal fluid where its concentration is much higher,” Toloue said. “Its measurement in serum/plasma was enabled by Quanterix’s highly sensitive single molecule array technology (Simoa), which digitizes immunoassays whereby molecules are individually counted.”
Quanterix expects to offer the test for clinical use in the United States in the second half of this year. “Additional clinical validation of the test is ongoing to support FDA clearance of the test, with submission expected in the first half of 2023,” Toloue said.
The FDA’s Breakthrough Therapy Designation comes on the heels of a large-scale, international study published in The Lancet Neurology, in which researchers from the University Hospital Basel and University of Basel leveraged Quanterix’technology to help establish a new method for clinicians to identify and interpret elevated values of sNfL in individual MS patients.
In that study, the researchers created a control group of participants with no evidence of central nervous system disease taking part in four cohort studies in Europe and North America. They tested the reference database in participants with MS in the Swiss Multiple Sclerosis Cohort (SMSC) and compared the association of sNfL Z scores with clinical and MRI characteristics recorded longitudinally to ascertain their respective disease prognostic capacity.
The researchers validated their findings in an independent sample of individuals with multiple sclerosis who were followed up in the Swedish Multiple Sclerosis registry.
In addition, the Simoa NfL assay was referenced in at least 20 studies presented at the American Academy of Neurology (AAN) 74th Annual Meeting, further validating the biomarker’s potential utility.
Research studies have shown the Simoa NfL test to be more sensitive and predictive of MS disease activity in relapsing remitting MS (RRMS) than MRI, according to Toloue. “Because the test is more sensitive, predictive, and can be given at more frequent intervals than MRI, it has the potential to provide a useful adjunctive tool with which optimize a treatment course, including deciding when to switch treatments,” he said.
The Breakthrough Device designation for this test “has the potential to help the multiple sclerosis community further advance the optimal use of NfL measurements in both research and clinical practice aimed at more effective therapeutic management of the disease for the millions of patients suffering from the condition,” said Mark S. Freedman, M.D., professor of neurology and director of Multiple Sclerosis Research at the Ottawa Hospital in Ottawa, Ontario, in the Quanterix news release.
The FDA’s Breakthrough Device designation is granted to products that have the potential to offer more effective diagnosis or treatment of life-threatening diseases with an unmet medical need.