The FDA has issued draft guidance outlining how minimal residual disease (MRD) and complete response (CR) can be used as primary endpoints to support accelerated approval of drugs and biologics for multiple myeloma. Under the proposal, sponsors may use MRD negativity—defined as the absence of detectable myeloma cells in bone marrow by highly sensitive flow cytometry or sequencing assays after a complete response—as a surrogate endpoint reasonably likely to predict long‑term clinical benefit. CR, including stringent complete response, can also serve as an accelerated approval endpoint if durability and adequate follow‑up are demonstrated.
The guidance recommends randomized trials as the preferred approach so MRD or CR can be evaluated alongside progression‑free and overall survival, but allows single‑arm designs in certain settings. It calls for validated MRD assays, prespecified statistical plans, robust data collection and concurrent confirmatory trials designed to verify clinical benefit after an accelerated approval is granted. The move follows a unanimous 2024 vote by the Oncologic Drugs Advisory Committee supporting MRD‑negative response as a surrogate endpoint for accelerated approval in myeloma and reflects pressure to find more sensitive efficacy measures as existing regimens already achieve high overall response rates. If finalized, the policy is expected to influence trial design, regulatory strategy and business valuations across late‑stage myeloma pipelines.
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