Pepaxti

Pepaxti, in combination with dexamethasone, is indicated for adult patients with multiple myeloma 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 drug received Orphan medicinal product designation on 19 March 2015 and a positive opinion for marketing authorisation on 23 June 2022.

In clinical trials, the overall confirmed response rate (ORR) for Pepaxti was observed to be between 29.1% and 33.8%. The median duration of response (DoR) ranged from approximately 3 months to 11 months. In a Phase 3 study (OP-103/OCEAN) involving patients with relapsed or refractory multiple myeloma, the median progression-free survival (PFS) for melflufen + dexamethasone was 6.83 months, compared to 4.93 months for pomalidomide + dexamethasone. The median overall survival (OS) was 20.24 months for melflufen + dexamethasone and 23.98 months for pomalidomide + dexamethasone.

Safety data from 495 patients indicated that 73.9% of patients experienced melflufen dose modifications, and 80.9% of patients had dose modifications overall. The median duration of treatment in the TSP study was 17.9 weeks, with a median of 3.0 completed treated cycles. 34.1% of patients in the TSP study remained on the study drug for at least 6 months.