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Pepaxto

melphalan flufenamide
Peptide-Drug ConjugateFDA Approved 2021Oncopeptides
Route
IV
Half-Life
~6.7 hrs
FDA Approved
2021
Manufacturer
Oncopeptides
1. Indications and Usage

Note: Initially approved for R/R MM; voluntarily withdrawn from US market in 2022. Included for reference.

2. Dosage and Administration

40 mg IV on Day 1 of each 28-day cycle with dexamethasone.

3. Dosage Forms and Strengths

Injection: 20 mg lyophilized powder in single-dose vial

4. Contraindications

None listed in the prescribing information.

5. Warnings and Precautions
  • Myelosuppression: Severe thrombocytopenia and neutropenia.
  • Infections: Serious and fatal infections.
  • Embryo-Fetal Toxicity: Can cause fetal harm.
6. Adverse Reactions
Most Common Adverse Reactions

Neutropenia (79%), Thrombocytopenia (76%), Anemia (43%), Fatigue (32%), Nausea (24%), Diarrhea (22%), Pyrexia (20%)

Neutropenia
79%
Thrombocytopenia
76%
Anemia
43%
Fatigue
32%
Nausea
24%
Diarrhea
22%
Pyrexia
20%

Consult the complete prescribing information for a comprehensive list of adverse reactions and their frequencies.

7. Drug Interactions

No formal drug interaction studies.

8. Use in Specific Populations
Pregnancy

Consult the full prescribing information for pregnancy-related considerations.

Lactation

Refer to prescribing information for lactation guidance.

Pediatric Use

Pediatric safety and efficacy information is detailed in the full label.

Hepatic/Renal Impairment

Dose modifications for organ impairment are specified in the complete prescribing information.

12. Clinical Pharmacology
Mechanism of Action

Melphalan flufenamide (melflufen) delivers alkylating agent melphalan preferentially to aminopeptidase-rich tumor cells. Intracellular cleavage releases melphalan for higher intratumoral concentrations.

Pharmacokinetics

Half-life (melflufen): <5 minutes; rapidly cleaved to melphalan. Melphalan half-life: ~1 hour.

14. Clinical Studies

Clinical efficacy and safety data supporting the approval are available in the full prescribing information and from the clinical trials listed below.

Pivotal Clinical Trials
Additional Resources
📋 All Melphalan Flufenamide Trials on ClinicalTrials.gov ↗ 📚 PubMed: Melphalan Flufenamide Clinical Trials ↗
FDA-Approved Tumor Types

Pepaxto has FDA-approved indications across the following cancer types covered on CancerDrugEvidence:

Multiple Myeloma
External Resources
📋 DailyMed Label ↗🏛️ FDA Drugs@FDA ↗🔬 ClinicalTrials.gov ↗📚 PubMed Pivotal Trials ↗
Important Notice: This page is intended as a navigational reference to the FDA-approved prescribing information for Pepaxto. It does not replace the full prescribing information. Healthcare professionals should consult the complete package insert available at DailyMed before making prescribing decisions. Patient-specific factors should always guide clinical decision-making.

Frequently Asked Questions

What is Pepaxto (melphalan flufenamide) approved for?

Pepaxto (melphalan flufenamide) is an FDA-approved oncology agent. Refer to the full prescribing information for complete indication details.

How is Pepaxto (melphalan flufenamide) administered?

Refer to the full prescribing information for dosing schedules, administration instructions, and dose modifications.

How does Pepaxto (melphalan flufenamide) work?

Melphalan flufenamide (melflufen) delivers alkylating agent melphalan preferentially to aminopeptidase-rich tumor cells. Intracellular cleavage releases melphalan for higher intratumoral concentrations.

What are the most common side effects?

Neutropenia (79%), Thrombocytopenia (76%), Anemia (43%), Fatigue (32%), Nausea (24%), Diarrhea (22%), Pyrexia (20%) Neutropenia 79% Thrombocytopenia 76% Anemia 43% Fatigue 32% Nausea 24% Diarrhea 22% Pyrexia 20%

← All TherapiesFull Prescribing Information ↗