European Medicines Agency´s (EMA) Committee for Medicinal Products for Human Use (CHMP), has adopted a positive opinion recommending marketing authorization approval (MAA) of melphalan flufenamide, also called melflufen, a drug commercialised by Oncopeptides with the name of Pepaxti®.
The positive opinion is based on data from the phase 2 HORIZON study and is supported by data from the randomized controlled phase 3 OCEAN study which was utilized as a confirmatory study. Melflufen is indicated, in combination with dexamethasone, for the treatment of adult myeloma patients who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.
“The recommendation for full approval of this drug by EMA is really good news for patients with triple class refractory disease, where the unmet medical need remains high and treatment options often are exhausted,” says Pieter Sonneveld, professor of Hematology at the Erasmus University Medical Center in Rotterdam, the Netherlands and principal investigator of the OCEAN study.
Oncopeptides intends to submit a type II variation in Q4 2022 to enable access to earlier lines of treatment for patients with relapsed refractory multiple myeloma (RRMM).
Read here the full press release here.