CHMP Backs Pepaxti Label Expansion to Third-Line Multiple Myeloma in Europe
核心洞察
The EMA's CHMP has adopted a positive opinion recommending Pepaxti plus dexamethasone for adults with multiple myeloma (搜索) after at least two prior lines of therapy.
The expanded indication would drop the current triple-class refractory requirement, moving Pepaxti from fourth-line-and-later use into third-line treatment.
Oncopeptides (搜索) estimates the third-line label could at least double Pepaxti's addressable European patient population and lengthen average treatment duration.
The European Medicines Agency (搜索)'s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending an extension of the therapeutic indication for Oncopeptides (搜索)' Pepaxti (melphalan flufenamide, also called melflufen) to earlier lines of therapy in multiple myeloma (搜索). The opinion, announced on September 17, 2026, will now be referred to the European Commission for a final decision, anticipated within 30 to 60 days.
Under the adopted opinion, the CHMP recommends expanding Pepaxti's indication, in combination with dexamethasone, to adult patients with multiple myeloma (搜索) who have received at least two prior lines of therapy, corresponding to third line and later, and whose disease is refractory to lenalidomide and the last line of therapy.
Shift Away From Triple-Class Refractoriness
Pepaxti is currently authorised in the European Union for adult patients who have received at least three prior lines of therapy and are triple-class refractory, effectively a fourth-line-and-later setting. That label requires disease refractory to at least one proteasome inhibitor, one immunomodulatory agent and one anti-CD38 (搜索) monoclonal antibody, with 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 three years from transplantation.
The proposed expansion would remove the triple-class refractory requirement and allow physicians to treat patients earlier in the disease course. Oncopeptides (搜索) estimates that moving into third-line treatment could at least double Pepaxti's addressable patient population in Europe, while average treatment duration is also expected to increase.
"The positive CHMP opinion paves the way for broader patient access and expanded commercial opportunities across Europe," said Sofia Heigis, CEO of Oncopeptides (搜索). "Expanding into the third line removes the triple-class refractory barrier, effectively doubles our addressable patient population and enables longer treatment duration."
OCEAN Trial Evidence
The recommendation is supported by clinical evidence from the Phase III OCEAN study, which evaluated efficacy and safety in patients with relapsed and refractory multiple myeloma (搜索). According to the company, earlier-line treatment allows patients to remain on therapy longer, with the average number of treatment cycles per patient in the third line estimated to double compared with the current fourth-line indication.
Regulatory and Commercial Sequence
The CHMP opinion follows a Type II variation application submitted to the EMA in June 2026 seeking the third-line indication. Oncopeptides (搜索) has also been building out its European commercial infrastructure. In May, it signed an agreement with SALUS (搜索) for distribution and commercialisation of Pepaxti across 11 Central and Eastern European markets, and Slovenia subsequently became the first country in the region to establish a list price for the medicine.
Upon EC approval, Oncopeptides (搜索) plans to begin local pricing and reimbursement discussions across Europe. In Germany, the company expects to submit its access dossier approximately one month after EC approval, allowing commercial sales under the country's existing regulatory framework.
Pepaxti has been granted Marketing Authorization in the European Union, the EEA countries Iceland, Liechtenstein and Norway, as well as in the UK. Oncopeptides (搜索) is a Swedish biotech company focused on research, development and commercialisation of targeted therapies for difficult-to-treat cancers, using its proprietary Peptide Drug Conjugate (PDC) platform to deliver cytotoxic agents selectively into cancer cells. The company is also developing compounds based on its PDC and SPiKE technology platforms.