Table 1 Use of RWE in FDA and EMA Drug Approvals in MM by Regulatory Review Pathway and Line of Therapy (January 2021–April 2025).
From: Utilization of real-world evidence in regulatory approvals for multiple myeloma therapies
Multiple myeloma therapies | Relevant FDA & EMA pathways/designationsa | Pivotal clinical trial(s) (NCT #) | Line of therapy approval | Total FDA/EMA approvals | |||
|---|---|---|---|---|---|---|---|
2 L + | 3 L + | 4 L + | 5 L + | ||||
Ciltacabtagene autoleucel | FDA: BTD, ODD, PR EMA: CMA, OMP | CARTITUDE-1 (NCT03548207) CARTITUDE-4 (NCT04181707) |
|
|
| 3 | |
Elranatamab | FDA: AA, BTD, ODD, PR EMA: CMA, OMP, PRIME | MAGNETISMM-3 (NCT04649359) |
| 1 | |||
Idecabtagene vicleucel | FDA: BTD, ODD EMA: CMA, OMP | KarMMa-3 (NCT03651128) |
|
|
| 3 | |
Linvoseltamab | FDA: AA, FT, ODD, PR EMA: CMA | LINKER-MM1 (NCT03761108) |
| 1 | |||
Melphalan flufenamideb | FDA: AA, ODD, PR EMA: OMP | HORIZON (NCT02963493) |
| 1 | |||
Talquetamab | FDA: AA, BTD, ODD, PR EMA: CMA, OMP, PRIME | MonumenTAL-1 (NCT04634552) |
|
| 2 | ||
Teclistamab | FDA: AA, BTD, ODD, PR EMA: CMA, OMP, PRIME | MajesTEC-1 (NCT04557098) |
| 1 | |||
Total | 1 | 1 | 5 | 5 | 12 | ||
- 1 L first-line, 1 L+ first-line or higher, 2 L+ second-line or higher, 3 L+ third-line or higher, 4 L+ fourth-line or higher, 5 L+ fifth-line or higher.
- FDA U.S. Food and Drug Administration, EMA European Medicines Agency, MM multiple myeloma, RWE real-world evidence, AA Accelerated Approval, BTD breakthrough therapy designation, ODD orphan drug designation, NCT national clinical trial, PR priority review, OMP orphan medicinal product, CMA conditional marketing authorization, PRIME priority medicines scheme, FT fast track.
- aNon-exhaustive and not inclusive of other therapeutic areas.
- bMelphalan flufenamide has been withdrawn from the U.S. market. FDA’s formal withdrawal of approval became effective on February 23, 2024.
Natural history study.
External control arm.
Natural history study.
External control arm.