ArticleTranslational oncology2022
The approval and withdrawal of melphalan flufenamide (melflufen): Implications for the state of the FDA.
Article in Translational oncology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 45 citations in OpenAlex.
- FDA-Approved Drugs Containing Amide Functionality in the Last Five Years (2021-2025): Pharmaceutical Use, Trends and Synthetic Approaches.Medicines (Basel, Switzerland) · 2026Review
- Clinical Benefit of Multiple Myeloma Drugs at Regulatory Approval in Brazil, Europe, and the USA: A Retrospective Cohort Study (2003-2024).Clinical drug investigation · 2026Article
- Tumor-Selective and Chemical Activation Strategies for Nitrogen Mustard Prodrugs.Chemistry (Weinheim an der Bergstrasse, Germany) · 2026Review
- Innovative Peptide Therapeutics in the Pipeline: Transforming Cancer Detection and Treatment.International journal of molecular sciences · 2025Review
- A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2024.Naunyn-Schmiedeberg's archives of pharmacology · 2025Review
- Trends in the research and development of peptide drug conjugates: artificial intelligence aided design.Frontiers in pharmacology · 2025Review
- Recent advances in targeted drug delivery systems for multiple myeloma.Journal of controlled release : official journal of the Controlled Release Society · 2024Review
- Dual-Action Therapeutics: DNA Alkylation and Antimicrobial Peptides for Cancer Therapy.Cancers · 2024Review
- An empirical analysis of overall survival in drug approvals by the US FDA (2006-2023).Cancer medicine · 2024Article
- Review
- Antibody-Drug Conjugates: The Dynamic Evolution from Conventional to Next-Generation Constructs.Cancers · 2024Review
- Enriching single-arm clinical trials with external controls: possibilities and pitfalls.Blood advances · 2023Article
- Peptide cargo administration: current state and applications.Applied microbiology and biotechnology · 2023Review
- Recent Advances in the Applications of Small Molecules in the Treatment of Multiple Myeloma.International journal of molecular sciences · 2023Review
- Peptide Drug Conjugates and Their Role in Cancer Therapy.International journal of molecular sciences · 2023Review
- Randomised controlled trials evaluating anticancer therapies in haematological cancers: an overview of global research activity.Ecancermedicalscience · 2023Article
- Value in Myeloma Care: Myth or Reality.Current hematologic malignancy reports · 2022Review
- The Role of Belantamab Mafodotin, Selinexor, and Melflufen in Multiple Myeloma.Current hematologic malignancy reports · 2022Review
- Learn from antibody-drug conjugates: consideration in the future construction of peptide-drug conjugates for cancer therapy.Experimental hematology & oncology · 2022Review
- Authors' reply: Perspective: The approval and withdrawal of melphalan flufenamide (melflufen): Implications for the state of the FDA.Translational oncology · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In October 2021, melphalan flufenamide (melflufen) was withdrawn from the US market for the treatment of multiple myeloma. The decision occurred based on results from a phase 3 randomized controlled trial (RCT) which showed numerically inferior overall survival, which previously led the FDA to halt all trials involving this drug. We highlight four issues raised by the approval fate of melflufen. First, the OCEAN trial was designed with a substandard control arm: negative results occurred despite this bias theoretically favoring the experimental arm. Second, a new compound, derived from a well-known drug, is not well fitting the accelerated pathway principles, unless being robustly tested against its parent drug. Third and four, allowing a new compound on the market, while there are known alternatives, and imminent confirmatory data, has the potential to harm patients while bringing earlier market share and profit to the company. While the FDA and the company should be commended for pushing a potentially dangerous product off the US market despite recent approval, yet a re-evaluation of regulatory processes is needed to ensure that cancer patients have timely access to effective medications while being protected against potentially detrimental ones.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.