C. Ola Landgren, Leader of Translational and Clinical Oncology Program/Experimental Therapeutics at Sylvester Comprehensive Cancer Center, shared a post on LinkedIn:
“Yesterday, the U.S. Food and Drug Administration (FDA) released a draft guidance for industry regarding the use of minimal residual disease (MRD) and complete response (CR) as primary endpoints in clinical trials evaluating drugs and biologics for the treatment of patients with multiple myeloma that would support their accelerated approval.
Here is the draft guidance document.
The draft guidance, entitled ‘Minimal Residual Disease and Complete Response in Multiple Myeloma: Use as Endpoints to Support Accelerated Approval,’ provides recommendations for sponsors who are designing multiple myeloma clinical trials that are seeking to use MRD or CR as an endpoint for an accelerated drug approval. In the guidance, MRD is determined using flow cytometry or next-generation sequencing (NGS) methods for patients who have already achieved a CR.
The use of MRD as an acceptable endpoint for the accelerated approval of drugs or biologics for patients with multiple myeloma was first accepted by the Oncology Drug Advisory Committee (ODAC) in April 2024, following an FDA-performed pooled analysis of data analyzing the relationship between minimal residual disease and long-term outcomes.
This Blood Cancer Discovery journal review paper (open access = FREE download for all readers) details the full ODAC journey on the roadmap on MRD as an early endpoint for accelerated drug approval in myeloma.”
You can read the full paper on Blood Cancer Journal.