Today the U.S. Food and Drug Administration permitted marketing of ClonoSEQ assay, a next-generation sequencing (NGS)-based test for minimal residual disease (MRD) in patients with acute lymphoblastic leukemia (ALL) or multiple myeloma.
"At the FDA, we are continuing to maximize opportunities for innovation that can improve patient outcomes," said FDA Commissioner Scott Gottlieb, M.D. "Today's approval is an important step forward for patients suffering from ALL and multiple myeloma.
Determining whether a patient has residual cancer cells remaining after treatment provides information on how well a patient has responded to therapy and how long remission may last.
Having a highly sensitive test available to measure minimal residual disease in ALL or multiple myeloma patients can help providers manage their patients' care. 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 see more and more laboratory-developed tests seek marketing authorization from the FDA. We're doing as much as we can to advance these opportunities for patients under our current authorities.
But we believe that to more fully unlock these innovations, we need to modernize the regulatory framework for all in vitro clinical tests. We put forward one such plan. We believe such an approach can promote the development of safe, effective technologies that have the greatest potential to help us diagnose, treat and cure disease."
B-Cell ALL is a rapidly progressing cancer that forms in the bone marrow and results in an increased number of abnormal white blood cells in the bloodstream and bone marrow.
Multiple myelomas are cancer that begins in plasma cells, a type of white blood cell. Malignant plasma cells accumulate in the bone marrow, crowding out the normal plasma cells that help fight infection. It is estimated that in 2018 approximately 6,000 people in the United States will be diagnosed with ALL and approximately 31,000 new cases of multiple myeloma will be diagnosed.
MRD is a general measure of the amount of cancer in the body (tumor burden), specifically the number of cancer cells that remain in a person's bone marrow, either during or after treatment. Measuring MRD provides a tool to detect very low levels of tumor burden.
MRD is useful to evaluate in patients who have responded to therapy when their tumor burden is below what can be detected with standard methods. The detection of MRD is associated with recurrence of the disease in those patients.
Currently, providers test for MRD using diagnostics called flow cytometry assays or polymerase chain reaction (PCR)-based assays. Those methods are usually capable of measuring MRD down to 1 in 10,000 or 1 in 100,000 cells.