At a glance
What is it—and why does it matter?
Melflufen is described as a peptide–drug conjugate prodrug designed to deliver the alkylating agent melphalan. With a median follow-up duration of 5.75 months, the overall survival curve had not reached the median in this melflufen–dexamethasone cohort.
Sources for this introduction: [1] [2]
This published profile is a reference in progress. Findings retain their source context; missing topics are marked below.
What is it?
A molecule, not a product name.
Identity
Melflufen is described as a peptide–drug conjugate prodrug designed to deliver the alkylating agent melphalan.
- Source records
- 6
- Independent studies
- 5
Evidence boundary: Applies only to the source-defined population, formulation, dose, and assessment period.
Exact passages, locators, and provenance
- supports · Source-backed record
“melflufen (melphalan flufenamide)”
Efficacy of melflufen in multiple myeloma with mutated or deleted TP53. · Abstract
pubmed:41437395:1a8db8601b79:1a8db8601b79 - supports · Source-backed record
“Melflufen, a first-in-class peptide-drug conjugate”
Optimizing the use of melflufen (melphalan flufenamide) in relapsed or refractory multiple myeloma: recommendations for clinical practice. · Abstract
pubmed:41207923:bdd9cc437c00:bdd9cc437c00 - supports · Source-backed record
“melflufen (melphalan flufenamide)”
Optimizing the use of melflufen (melphalan flufenamide) in relapsed or refractory multiple myeloma: recommendations for clinical practice. · Abstract
pubmed:41207923:bdd9cc437c00:bdd9cc437c00 - supports · Source-backed record
“melflufen (a peptide-drug conjugate pro-drug of melphalan)”
Exceptional long-term responses from OCEAN and HORIZON trials: melflufen-dexamethasone as an expansion of treatment options for relapsed/refractory multiple myeloma in the era of new immunotherapies? · Abstract
pubmed:41065854:0ad5255cf0e7:0ad5255cf0e7 - supports · Source-backed record
“Melflufen (melphalan-flufenamide) plus dexamethasone”
Positioning of Melflufen in Heavily Pretreated RRMM Patients: Real-World Evidence in a Rapidly Evolving Therapeutic Landscape. · Abstract
pubmed:41813608:eed0813f961b:eed0813f961b - supports · Source-backed record
“Melflufen (melphalan flufenamide)”
Melflufen in Multiple Myeloma: Clinical Limitations, Biological Rationale, and Future Perspectives. · Abstract
pubmed:42192911:29849c8a34c3:29849c8a34c3
How does it work?
Target, response, and disposition.
This profile does not yet contain a published summary for this topic. This is a coverage gap, not evidence that no research exists.
What has been studied?
What the evidence says.
Study findings
With a median follow-up duration of 5.75 months, the overall survival curve had not reached the median in this melflufen–dexamethasone cohort.
- Reported result
- median overall survival was not reached
- Source records
- 1
- Independent studies
- 1
Evidence boundary: Applies only to the source-defined population, formulation, dose, and assessment period.
Exact passages, locators, and provenance
- supports · Source-backed record
“At a median follow-up of 5.75 months, median overall survival was not reached.”
Real-World Outcomes of Melflufen Plus Dexamethasone in Heavily Pretreated Relapsed/Refractory Multiple Myeloma After Exposure to Immunotherapies. · RESULTS
pubmed:42270486:2c8dd0b57466:2c8dd0b57466
Study findings
Time-to-event outcome for melflufen–dexamethasone showed a median progression-free survival of 4.8 months, with a 95% confidence interval from 2.5 months to not reached.
- Reported result
- 4.8 months (95% CI: 2.5-not reached)
- Source records
- 1
- Independent studies
- 1
Evidence boundary: Applies only to the source-defined population, formulation, dose, and assessment period.
Exact passages, locators, and provenance
- supports · Source-backed record
“Median progression-free survival was 4.8 months (95% CI: 2.5-not reached).”
Real-World Outcomes of Melflufen Plus Dexamethasone in Heavily Pretreated Relapsed/Refractory Multiple Myeloma After Exposure to Immunotherapies. · RESULTS
pubmed:42270486:2c8dd0b57466:2c8dd0b57466
Risks and interactions
Risks, organized for scanning.
This profile does not yet contain a published summary for this topic. This is a coverage gap, not evidence that no research exists.
Products and regulatory status
Same ingredient. Different records.
This profile does not yet contain a published summary for this topic. This is a coverage gap, not evidence that no research exists.
Administration context
The practical clinical context.
This profile does not yet contain a published summary for this topic. This is a coverage gap, not evidence that no research exists.
Research status + gaps
What still needs better answers?
- No publishable claim yet for: administration, contraindication, interaction, mechanism, regulatory, safety
- 62 compiled claim(s) withheld by automated assurance: assurance_score_below_0.58 (25), assurance_score_below_0.72 (28), current_regulatory_source_required (12), extraction_ambiguity (54), high_risk_requires_regulatory_or_two_independent_sources (37), no_direct_support (53)
These are the limits of this profile, not an exhaustive list of scientific uncertainties.
References + discovery
Open the records yourself.
- Exceptional long-term responses from OCEAN and HORIZON trials: melflufen-dexamethasone as an expansion of treatment options for relapsed/refractory multiple myeloma in the era of new immunotherapies? ↗
pubmed · published 2025-10-09 · retrieved 2026-08-28T12:35:21Z
- Optimizing the use of melflufen (melphalan flufenamide) in relapsed or refractory multiple myeloma: recommendations for clinical practice. ↗
pubmed · published 2025-11-01 · retrieved 2026-08-28T12:35:21Z
- Efficacy of melflufen in multiple myeloma with mutated or deleted TP53. ↗
pubmed · published 2025-12-23 · retrieved 2026-08-28T12:35:21Z
- Positioning of Melflufen in Heavily Pretreated RRMM Patients: Real-World Evidence in a Rapidly Evolving Therapeutic Landscape. ↗
pubmed · published 2026-06-01 · retrieved 2026-08-28T12:35:21Z
- Melflufen in Multiple Myeloma: Clinical Limitations, Biological Rationale, and Future Perspectives. ↗
pubmed · published 2026-05-11 · retrieved 2026-08-28T12:35:21Z
- Real-World Outcomes of Melflufen Plus Dexamethasone in Heavily Pretreated Relapsed/Refractory Multiple Myeloma After Exposure to Immunotherapies. ↗
pubmed · published 2026-08-01 · retrieved 2026-08-28T12:35:21Z
Publication history and provenance
Version 2 · Automated assessment · 2026-08-28T12:35:21Z
d4d11c01ea759772901950440ca6f6bb89d126281eac09ce2711e958d7cb9820