On September 17, 2026, Europe's main drug-review committee recommended widening the approval of Pepaxti, a multiple myeloma treatment, so that patients can receive it earlier in their course of care. The same Phase 3 trial behind that recommendation is the reason the drug was pulled from the United States market two years before.
The committee is the CHMP, the scientific panel that advises the European Medicines Agency. Its positive opinion was announced by the drug's maker Oncopeptides ↗. It would let doctors prescribe Pepaxti to adults who have already tried at least two prior lines of treatment. Those patients must also have disease that has stopped responding to lenalidomide and to their most recent therapy. Today the European label restricts the drug to patients who have exhausted at least three prior lines and are resistant to three whole classes of myeloma drugs. In practice the change moves the drug from a last-resort option to something a doctor can reach for a step or two sooner. The company says that roughly doubles the number of European patients who would qualify.
The drug's active ingredient is melflufen, a peptide-drug conjugate. That means a small chemotherapy payload is bolted onto a short peptide carrier so the toxic part stays quiet until it reaches the tumor. Myeloma cells are unusually rich in enzymes called aminopeptidases. Those enzymes cut the carrier loose and release a classic chemotherapy, melphalan, right where it does the most damage. The design is meant to concentrate the poison in cancer cells and spare healthy tissue.
The problem is the trial that both regulators are reading. The OCEAN study ↗00343-2/fulltext) tested melflufen plus dexamethasone against pomalidomide plus dexamethasone, a standard comparison drug, in patients whose myeloma had returned. Melflufen delayed the cancer from worsening: median progression-free survival was 6.8 months versus 4.9 months, meaning the disease stayed stable about two months longer. But on the measure that matters most, how long patients actually lived, the drug lost. Median overall survival was 19.8 months on melflufen against 25 months on the comparison, so patients on the peptide conjugate lived roughly five months less. One subgroup fared clearly worse. Patients who had received a stem-cell transplant less than three years earlier lived a median of 15.7 months on melflufen versus 28.7 months on the comparison. That is close to double the risk of death.
That survival signal is what split the two regulators. A US advisory panel voted 14 to 2 against the drug in September 2022, and the FDA formally withdrew Pepaxto ↗ from the American market in February 2024. Europe kept its narrower approval in place and is now proposing to broaden it.
Most coverage of the CHMP news has led with the commercial framing. Oncopeptides chief executive Sofia Heigis called the opinion a step that "paves the way for broader patient access and expanded commercial opportunities across Europe." What the announcements tend to skip is that the earlier-line expansion pushes the drug toward exactly the kind of patient the OCEAN data flagged. Patients treated sooner are more likely to have had a recent transplant, and that recent-transplant group is the one where survival was worst. The trial that justifies moving the drug earlier is also the trial that recorded harm in the population most likely to be treated earlier.
None of this is settled by the CHMP opinion. A positive recommendation is advice, not a license. The European Commission still has to issue a formal decision, usually within one to two months. The wording of that final indication can narrow what the committee proposed. The gap worth watching is not whether the drug works at holding disease back for a while. It clearly does. The gap is whether living slightly longer without progression is worth a shorter life overall, and whether Europe and the United States can keep reading the same numbers to opposite conclusions.