EMA committee recommends melflufen label expansion to third-line myeloma treatment
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On September 17, 2026, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended extending the approved use of melphalan flufenamide (melflufen, Pepaxti®) to third-line myeloma treatment.
The CHMP is the European Medicines Agency’s (EMA) committee responsible for evaluating medicines and making recommendations on whether they should be approved for use in the European Union (EU). A positive recommendation from the CHMP still requires formal approval by the European Commission before it can be implemented, a process that typically takes a few months.
Melflufen is a medicine called a peptide drug conjugate. It is designed to deliver the active chemotherapy agent melphalan into myeloma cells, where it helps damage and kill them.
The CHMP recommendation would allow melflufen, in combination with dexamethasone, to be used in myeloma patients who have received at least two prior lines of therapy and whose disease is refractory to lenalidomide and their last line of treatment.
Melflufen is currently authorised in the EU for myeloma patients who have received at least three prior lines and whose disease is refractory to a proteasome inhibitor (e.g. bortezomib or carfilzomib), an immunomodulatory agent (e.g. lenalidomide or pomalidomide) and an anti-CD38 monoclonal antibody (e.g. daratumumab or isatuximab).
The recommendation is based on data from the phase 3 OCEAN trial. This study showed that melflufen plus dexamethasone delayed myeloma progression compared with pomalidomide plus dexamethasone, although it did not significantly improve overall survival in the overall trial population.
If approved, this would provide another treatment option for third-line myeloma patients in the EU. Following European Commission approval, treatment availability will depend on pricing, reimbursement and access decisions in individual European countries.