The FDA authorized the next-generation sequencing (NGS)–based ClonoSEQ assay for the measurement of minimal residual disease (MRD) in patients with acute lymphocytic leukemia (ALL) or multiple myeloma (MM). This is the first NGS-based test approved for this purpose.
The FDA’s approval was based on results from a retrospective analysis of three separate studies comprising 273 patients with ALL, 323 patients with MM, and 706 patients with MM, respectively. In patients with ALL, negative MRD results detected by the ClonoSEQ assay were correlated with higher event-free survival rates; in patients with MM, negative results were associated with higher progression-free and disease-free survival rates (p values not provided).
The ClonoSEQ assay identifies and quantifies gene sequences extracted from patient DNA and can measure MRD down to 1 in 1 million cells (<10-6), while previously available methods measured MRD via flow cytometry or polymerase chain reaction–based assays down to 1 in 10,000 (<10-4) or 1 in 100,000 cells (<10-5).
The FDA reviewed the ClonoSEQ assay through the de novo premarket review pathway, which is designed for novel, low- to moderate-risk devices of a new type.
“Having a highly sensitive test available to measure MRD in [patients with] ALL or MM can help providers manage their patients’ care,” FDA Commissioner Scott Gottlieb, MD, said in a press release announcing the approval. “The FDA is applying novel regulatory approaches to make sure that these rapidly evolving NGS tests are accurate and reliable. At the same time, we’re seeing more and more laboratory-developed tests seek marketing authorization from the FDA. … We believe that to more fully unlock these innovations, we need to modernize the regulatory framework for all in vitro clinical tests.”
Source: FDA press release, September 28, 2018.