Melphalan Flufenamide (Pepaxto) – 5L+ Multiple Myeloma

Full Efficacy & Safety Summary Table

IndicationIn combination with dexamethasone for relapsed MM following at least 4 prior lines of therapy
Registrational TrialAccelerated Approval. Phase 2 Single Arm OCEAN trial (n=157) (NCT03151811)
Regimen40 mg administered intravenously over 30 minutes on Day 1 of each 280 day cycle until disease progression or until acceptable toxicity
Efficacy OutcomesORR: 23.70%                                       OS: 9.1 months
DOR: 4.2 months                                PFS: 3.8 months
Safety OutcomesGr 3/4 ARs (≥2%): Pneumonia 11%, Fatigue 6%, Respiratory Tract Infection 5%

Dose Reduction: 27%
Discontinuation: 22%

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