Today, the US Food and Drug Administration has authorized the marketing of ClonoSEQ assay, a next-generation test (NGS) based on minimal residual disease (MRD) in patients with acute lymphoblastic leukemia (ALL) or multiple myeloma. MRD is a measure of the amount of cancer cells that remain in the bone marrow of a person.
"At the FDA, we continue to maximize the chances of innovation that can improve patient outcomes," said FDA Commissioner Scott Gottlieb, MD. "Today's approval is a major step forward for patients suffering from ALL and multiple myeloma, and residual cancer cells remaining after treatment provide information on how well a patient has responded to treatment and how long remission can last. Having a very sensitive test available to measure minimal residual disease in ALL or multiple myeloma patients can help healthcare providers manage their patients, and the FDA applies new regulatory approaches to ensure that these rapidly evolving NGS tests are accurate. and at the same time we see that more and more tests developed by laboratories are looking for the FDA's marketing authorization.We can promote these opportunities for patients under our current authorities, but we believe we should better unlock these innovations, we to modernize the regulatory framework for all in vitro clinical trials such a plan. We believe that such an approach can promote the development of safe, effective technologies that have the greatest potential to help us diagnose, treat and cure diseases. "
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 myeloma is a cancer that starts in plasma cells, a type of white blood cell. Malignant plasma cells accumulate in the bone marrow and displace the normal plasma cells that help fight infection. It is estimated that about 6,000 people in the United States will receive a diagnosis of ALL in 2018 and about 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), in particular the number of cancer cells that remain in the bone marrow of a person during or during the treatment. Measuring MRD provides a tool to detect very low levels of tumor load. MRD is useful for evaluating patients who have responded to therapy when their tumor burden is lower than what can be detected with standard methods. The detection of MRD is associated with a recurrence of the disease in those patients. Currently, providers are testing for MRD using diagnostic tests, flow cytometry tests or polymerase chain reaction (PCR) -based tests. These methods are usually able to measure MRD to 1 in 10,000 or 1 in 100,000 cells.
The ClonoSEQ assay is an in vitro diagnosis using multiplex PCR and NGS to identify and quantify certain gene sequences in bone marrow extracted DNA from patients with ALL or multiple myeloma. The ClonoSEQ test measures the amount of MRD and can detect MRD at levels of less than 1 in 1 million cells. This is a single-site test collected by the patient's provider and sent to Adaptive Biotechnologies Corporation for evaluation.
The FDA evaluated data to demonstrate clinical validity from a retrospective analysis of samples obtained from three previous clinical trials involving 273 patients with ALL, an ongoing study in 323 patients with multiple myeloma and a study in 706 patients with multiple myeloma. For patients with ALL, the ClonoSEQ assay was used to determine MRD at different disease burden thresholds to demonstrate that the MRD level correlated with event-free survival time, after treatment, that the patient remains free of certain complications or events. Patients whose MRD-negative ClonoSEQ test result had a longer event-free survival, while patients with higher MRD test results had lower event-free survival rates. For patients with multiple myeloma, the ClonoSEQ assay showed similar associations with progression-free survival, the duration during and after the treatment of a disease in which a patient lives with the disease, but it does not get worse, and disease-free survival time after the primary treatment a cancer ends, that the patient survives without signs or symptoms of that cancer.
The FDA assessed the ClonoSEQ test via the de novo premarket review route, a regulatory path for new, low to medium risk devices of a new type. Together with this authorization, the FDA sets criteria, called special checks, that clarify the agency's expectations by guaranteeing the accuracy, reliability and effectiveness of tests intended to help measure MRD to reduce the change in disease burden during and after the treatment. These special checks, when met together with general controls, provide reasonable assurance of safety and effectiveness for these tests. This action also creates a new regulatory classification, which means that subsequent devices of the same type with the same intended use can go through the FDA 510 (k) process, allowing devices to obtain marketing authorizations through substantial equivalence with a predicate device. show.
The FDA has granted a marketing authorization for the ClonoSEQ test for adaptive biotechnologies.