The European Commission has approved marketing authorisation (e.g. a license) for melphalan flufenamide (also called melflufen), a drug marketed in Europe by Oncopeptides with the name of Pepaxti®. This follows the previous positive recommendation from the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP).
Melflufen is a new drug for the treatment of myeloma, known as a pepitide drug conjugate. It is a derivative of the chemotherapy drug melphalan, which is frequently used in stem cell transplantation.
Melflufen is specifically approved in combination with dexamethasone for patients with refractory myeloma, who have received at least three prior lines of therapy and are refractory to at least one proteasome inhibitor (PI – such as bortezomib), one immunomodulatory drug (IMiD – such as lenalidomide) and one anti-CD38 monoclonal antibody (such as daratumumab). To be eligible to receive melflufen patients who have received prior autologous stem cell transplantation, the time to progression should be at least three years from transplantation.
The approval is based on data from the Phase II HORIZON clinical trial and supported by data from the randomised controlled Phase III OCEAN clinical trial as confirmatory study.
The European marketing authorisation is valid in all EU member states, as well as in Iceland, Lichtenstein, and Norway. The approval means that melflufen is safe and effective for use in myeloma patients, however, to become available and accessible to patients it requires a funding decision by national healthcare systems. The company is planning their approach for bringing the product to market in Europe towards the end of 2022.
For further information, you can read the company press release here.