Pepaxti Approved to Treat Resistant Multiple Myeloma in Europe
Melflufen for adults with refractory disease after three lines of treatment
The European Commission has approved Pepaxti (melphalan flufenamide, also called melflufen), a medicine by Oncopeptides to be used in combination with dexamethasone, making it available to adults with hard-to-treat multiple myeloma in Europe.
The approval came after the Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency, gave its positive opinion on Pepaxti.
It is valid for all European Union (EU) countries as well as for Iceland, Liechtenstein, and Norway. The company is also working to launch the medicine in Germany toward the end of this year.
“The approval of Pepaxti in Europe is foundational for Oncopeptides, and brings excellent news for patients,” Jakob Lindberg, the company’s CEO, said in a press release.
Multiple myeloma occurs when plasma cells, a type of white blood cell, become cancerous and grow out of control in the bone marrow, the spongy tissue inside bones. Plasma cells fill the bone marrow, leaving no room for healthy blood cells to be made, and they can also damage the bone itself.
Melflufen helps address ‘high unmet need’ of refractory multiple myeloma
Pepaxti is indicated, in combination with dexamethasone (a corticosteroid), to treat adults with multiple myeloma who have received at least three prior lines of treatment.
Their disease should be refractory to at least one proteasome inhibitor, one immunomodulator, and one CD38 inhibitor, and to have progressed during or after the last line of treatment. The treatment for disease progression in patients who underwent an autologous stem cell transplant needs to be three or more years after transplantation.
“Despite the introduction of novel therapies, patients with triple class refractory disease have a high unmet medical need, since their treatment options ultimately become exhausted,” Lindberg said. Triple class refractory disease means the multiple myeloma has remained resistant (refractory) to at least three lines of treatment.
Pepaxti consists of melphalan, a medicine already approved for multiple myeloma, coupled to flufenamide. It works by rapidly delivering a cancer-killing agent — an alkylating peptide — to malignant cancer cells that contain high levels of aminopeptidases, which are enzymes that break down peptides (small proteins).
In this way, the therapy is designed to preferentially target and destroy cancer cells containing excessive amounts of these enzymes, while healthy cells are spared.
The EU decision was supported by the Phase 2 HORIZON clinical trial (NCT02963493), in which Pepaxti plus dexamethasone was found to be safe and bring about strong and long-lasting responses in adults with hard-to-treat multiple myeloma.
These findings were confirmed in OCEAN, a Phase 3 clinical trial (NCT03151811) that compared Pepaxti plus dexamethasone to Pomalyst (pomalidomide) plus dexamethasone in adults with multiple myeloma and two to four prior lines of treatment.
Oncopeptides also announced plans to request a change to the recent EU approval that could enable relapsed and refractory patients to access Pepaxti as an earlier line of treatment.
Pepaxti is currently not being marketed in the U.S., the company announced in March, due to differences in OCEAN trial findings between Oncopeptides and the U.S. Food and Drug Administration.