Oncopeptides’ Pepaxti (melflufen) has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), recommending an extension of its European indication to include third-line treatment of multiple myeloma.
The recommendation covers Pepaxti in combination with dexamethasone for adults with multiple myeloma who have received at least two prior lines of therapy and whose disease is refractory to lenalidomide and their most recent treatment.
The opinion will now be referred to the European Commission (EC), which is expected to make a final decision within 30-60 days, according to Oncopeptides.
Pepaxti is currently authorised in the EU for patients who have received at least three prior lines of therapy and are refractory to a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody — effectively a fourth-line-and-later setting.
The proposed label expansion would therefore allow treatment to begin earlier in the disease course and remove the current requirement for patients to be triple-class refractory.
Oncopeptides said the recommendation is supported by clinical evidence from the Phase III OCEAN study in relapsed and refractory multiple myeloma.
The company estimates that moving into third-line treatment could at least double Pepaxti’s addressable patient population in Europe, while average treatment duration is also expected to increase.
The development follows a series of regulatory and clinical activities around Pepaxti during 2026.
In June, Oncopeptides submitted a Type II variation application to the European Medicines Agency (EMA) seeking the third-line indication.
The company has also been expanding its European commercial infrastructure.
In May, it signed an agreement with SALUS for distribution and commercialisation of Pepaxti across 11 Central and Eastern European markets, while Slovenia subsequently became the first country in the region to establish a list price for the medicine.
If the EC approves the label extension, Oncopeptides plans to begin local pricing and reimbursement discussions across Europe.
In Germany, the company said it expects to submit its access dossier approximately one month after EC approval, allowing commercial sales under the country’s existing regulatory framework.
"The positive CHMP opinion paves the way for broader patient access and expanded commercial opportunities across Europe,” says Sofia Heigis, CEO of Oncopeptides.
"Expanding into the third line removes the triple-class refractory barrier, effectively doubles our addressable patient population and enables longer treatment duration."
If approved by the European Commission, this broader label would significantly unlock potential beyond our current indication and support our path to cash-flow positivity during 2027.
