The Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) has granted a positive opinion on the application for an extended marketing authorisation (i.e. a license) for melphalan flufenamide (Pepaxti), also called melflufen.

Melflufen, in combination with dexamethasone,  is currently licenced in the EU for treating myeloma patients who have received at least three prior lines of therapy and are refractory to at least one proteasome inhibitor (such as bortezomib or carfilzomib), one immunomodulatory agent (such as lenalidomide or pomalidomide) and one anti-CD38 monoclonal antibody (such as daratumumab).

According to the new decision, the CHMP now recommends an expansion of the licensed indication to include the use of melflufen in earlier lines of treatment – two or more lines of therapy. The extension also covers a new type of administration for melflufen, known as peripheral administration. Peripheral administration delivers melflufen through peripheral rather than central veins, a less invasive way of administration.

 
The recent CHMP decision is based on the findings from the OCEAN trial. In the trial, researchers compared the efficacy of melflufen plus dexamethasone vs pomalidomide and dexamethasone in patients with relapsed or refractory myeloma.

The trial included 495 patients who had received two to four previous lines of therapy, including lenalidomide and a proteasome inhibitor, and their disease was refractory to lenalidomide.

The results showed that melflufen plus dexamethasone demonstrated a better progression-free survival (PFS) compared to pomalidomide plus dexamethasone. The median PFS was 6.8 months in the melflufen group and 4.9 months in the pomalidomide group.

The opinion from the CHMP will now be sent to the European Commission for a final decision. This usually happens within 60 days of the CHMP opinion. If the Commission approves, the decision means doctors across the EU can prescribe the drug. However, it first requires funding approval from national health authorities for patients to receive it.

If you have any questions or comments on this decision, please email info@mpeurope.org