CHMP recommends expanding the use of melflufen — to include patients with disease progression after at least two prior lines of therapy, and those with lenalidomide-refractory myeloma.
On Thursday, September 17, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending Pepaxti (melflufen) to be used in earlier lines of treatment for multiple myeloma.
In a press release, Oncopeptides stated that the CHMP recommendation would expand melflufen’s use, to include patients whose multiple myeloma has progressed after at least two prior lines of therapy (third line and later) and whose disease is refractory to lenalidomide and the most recent line of therapy. Melflufen would be used in combination with dexamethasone.
The CHMP opinion will now be referred to the European Commission (EC), which makes the final decision on marketing authorization in the European Union. A decision is anticipated within 30 to 60 days.
According to a Journal of Clinical Medicine published review, melflufen (or melphalan flufenamide) is a first-in-class peptide–drug conjugate (PDC) that targets aminopeptidases to rapidly release alkylating agents inside tumor cells. Its distinct mechanism of action has supported its development as a third-line treatment for multiple myeloma, including in patients without prior alkylator therapy and those previously exposed to melphalan.
Melflufen is currently approved in the EU for adults with multiple myeloma who have received at least three prior lines of therapy and whose disease is triple-class refractory — meaning the current indication applies from the fourth line of therapy onward. The proposed change would move treatment into the third line and remove the triple-class refractory requirement.
The recommendation is based on clinical evidence from the Phase 3 OCEAN trial, which evaluated melflufen in patients with relapsed and refractory multiple myeloma (RRMM) and provided evidence on its efficacy and safety.
According to Oncopeptides, treatment earlier in the disease course is expected to allow patients to remain on melflufen for longer. The company estimates that the average number of treatment cycles per patient could approximately double in third-line treatment compared with its current fourth-line indication.
If the European Commission approves the expanded indication, Oncopeptides plans to begin pricing and reimbursement discussions in European markets.
In Germany, the company said existing regulatory frameworks would allow commercial sales under the expanded indication after submission of an access dossier, which it plans to submit approximately one month after EC approval.
The proposed indication remains subject to the European Commission’s final decision. Oncopeptides also said the expanded indication could broaden the number of patients eligible for melflufen.
References:
CHMP issues positive opinion of label expansion of Pepaxti to third-line treatment. Oncopeptides press release. September 17, 2026.
Schjesvold, F., Robak, P., Pour, L., Aschan, J., & Sonneveld, P. (2020). OCEAN: a randomized Phase III study of melflufen + dexamethasone to treat relapsed refractory multiple myeloma. Future Oncology, 16(11), 631–641. https://doi.org/10.2217/fon-2020-0024
Mateos, M.-V., Bladé, J., Bringhen, S., Ocio, E. M., Efebera, Y., Pour, L., Gay, F., Sonneveld, P., Gullbo, J., & Richardson, P. G. (2020). Melflufen: A Peptide–Drug Conjugate for the Treatment of Multiple Myeloma. Journal of Clinical Medicine, 9(10), 3120. https://doi.org/10.3390/jcm9103120