Last reviewed 2026-04-20 · How we verify

Pepaxto (MELPHALAN FLUFENAMIDE)

Pepaxto (MELPHALAN FLUFENAMIDE) is a small molecule alkylating drug originally developed by ONCOPEPTIDES AB. It is used to treat relapsed or refractory multiple myeloma, a type of blood cancer. Pepaxto works by interfering with the growth and survival of cancer cells. It is a patented medication, with its commercial status currently owned by ONCOPEPTIDES AB. Key safety considerations include the potential for severe side effects, such as myelosuppression and hepatotoxicity.

At a glance

Approved indications

• Relapsed or refractory multiple myeloma

Common side effects

No common side effects on file.

Key clinical trials

• Melflufen for Elderly Patients With Relapsed Myeloma (PHASE2)
• A Study of Melphalan Flufenamide (Melflufen)-Dex or Pomalidomide-dex for RRMM Patients Refractory to Lenalidomide (PHASE3)
• Study of Melflufen (Melphalan Flufenamide) in Combination With Daratumumab in Relapsed-Refractory Multiple Myeloma (PHASE3)
• PK of Melphalan During Treatment With Melflufen and Dexamethasone in Patients With RRMM and Impaired Renal Function (PHASE2)
• A PK, Safety and Tolerability Study of Peripheral and Central Infusion of Melflufen in RRMM Patients (PHASE2)
• Study of Melphalan Flufenamide (Melflufen) + Dex With Bortezomib or Daratumumab in Patients With RRMM (PHASE1,PHASE2)
• A Study of Melphalan Flufenamide (Melflufen) Plus Dexamethasone in Patients With Relapsed or Refractory Multiple Myeloma (PHASE2)
• A Clinical Study of Melphalan Flufenamide (Melflufen) and Dexamethasone for Patients With Immunoglobulin Light Chain (AL) Amyloidosis (PHASE1)

Patents

Primary sources

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