FDA Issues Draft Guidance on MRD and CR Endpoints for Accelerated Multiple Myeloma Drug Approvals
On January 20, 2026, the FDA released draft guidance allowing minimal residual disease (MRD) negativity and complete response (CR) as primary endpoints for accelerated approval of multiple myeloma drugs.234
MRD is defined as less than 1 myeloma cell per 1 million bone marrow cells using flow cytometry or next-generation sequencing in patients achieving CR.245
The guidance addresses challenges with traditional endpoints like overall response rate, enabling faster approvals especially for earlier treatment lines.13
Following unanimous ODAC endorsement in April 2024 based on pooled analyses linking MRD to improved PFS and OS.236
No direct praise from Johnson & Johnson (J&J) found in recent sources; guidance praised by experts like Ola Landgren and Multiple Myeloma Research Foundation.3
Accelerated approvals require confirmatory trials verifying benefit with PFS or OS endpoints.26
Sources:
1. https://www.thepharmaletter.com/pharma-news/fda-issues-draft-guidance-for-multiple-myeloma-drug-trials
2. https://ascopost.com/news/january-2026/fda-releases-draft-guidance-for-use-of-mrd-and-cr-as-primary-endpoints-in-multiple-myeloma-trials/
3. https://www.fiercebiotech.com/biotech/fda-outlines-draft-policy-mrd-complete-response-accelerated-approvals-multiple-myeloma
4. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/minimal-residual-disease-and-complete-response-multiple-myeloma-use-endpoints-support-accelerated
5. https://www.ajmc.com/view/fda-draft-guidance-proposes-cr-mrd-to-accelerate-mm-drug-approval
6. https://www.cancernetwork.com/view/fda-issues-guidance-on-mrd-cr-outcomes-for-multiple-myeloma-approvals