European Commission extends Pepaxti label to third-line multiple myeloma treatment

Oncopeptides AB announced that the European Commission has formally approved the Type II variation application for Pepaxti (melflufen), according to news-medical.net. The marketing authorization now covers adults with multiple myeloma who have received at least two prior lines of therapy (third line and later) and whose disease is refractory to lenalidomide and to the last line of therapy news-medical.netnews-medical.netnews-medical.netOpen source ↗. The decision formally confirms the positive opinion of the CHMP of the European Medicines Agency.

The indication was previously limited to the fourth-line setting. Broadening it to third-line therapy eliminates the requirement for triple-class refractoriness. This allows physicians to prescribe Pepaxti in earlier lines of therapy.

Clinical data behind the label

The expanded indication rests on data from the Phase 3 OCEAN trial and on safety data from several clinical trials and real-world use. These evaluated Pepaxti plus dexamethasone in relapsed and refractory multiple myeloma (RRMM). According to the company, the data set established efficacy and safety in this setting and confirms that earlier use can enable longer disease control and prolonged treatment duration.

The company states that, as shown in OCEAN, patients treated in earlier lines stay on therapy longer. In its account, this doubles the average number of treatment cycles per patient in third line compared with fourth line. The company also states that removing the triple-class refractoriness requirement doubles the number of eligible patients across Europe.

Launch plans in Europe

In Germany, Oncopeptides plans to submit its access dossier within approximately one month. According to the company, this permits commercial sales in the third-line setting while benefit assessment and pricing negotiations are underway. Germany currently uses a "basket" approach that averages modern third-line therapies, which the company describes as a comparative price anchor.

Formal pricing and reimbursement procedures will proceed in other key markets, including Spain and Italy, following local submission schedules. The company is also investigating how the new label could be used in additional markets.

CEO Sofia Heigis said the existing commercial footprint allows Pepaxti to be scaled cost-effectively in key markets, supporting the company's ambition to become cash flow positive during 2027 news-medical.netnews-medical.netnews-medical.netOpen source ↗.