FDA proposes new endpoints for multiple myeloma drug trials

The FDA issued draft guidance Jan. 21 proposing the use of minimal residual disease and complete response as primary endpoints to support accelerated approval of drugs for multiple myeloma.

The draft guidance outlines recommendations for trial design, statistical methods and assay validation for sponsors using these endpoints in clinical studies. It builds on pooled trial data presented at the April 12, 2024, meeting of the Oncology Drug Advisory Committee, where members unanimously agreed that MRD was an acceptable endpoint for accelerated approval.

Multiple myeloma accounts for 18% of all hematologic malignancies in the U.S., according to the agency. While overall response rates to new therapies now exceed 60-70% in relapsed or refractory disease and 90% in newly diagnosed cases, the FDA said these gains have made it increasingly difficult to demonstrate statistical differences using overall response rates alone.

MRD and complete response are both recognized prognostic biomarkers correlated with longer progression-free and overall survival. The draft guidance clarifies how each may be incorporated into future registration trials.

Comments on the draft guidance are due by March 23, 2026.