Oncopeptides — Pepaxti EMA EPAR variation assessment report (EMEA/H/C/005681, 2022)
Oncopeptides · 2022 · EMA EPAR variation assessment report
Report summary
This report summarizes a Type II variation application for Pepaxti (melphalan flufenamide) to expand its indication for relapsed or refractory multiple myeloma (RRMM) patients. The application is based on final results from the Phase 3 OCEAN study (OP-103) and supportive data from the Phase 2 HORIZON study (OP-106). The proposed indication targets adult patients who have received at least two prior lines of therapy and are refractory to lenalidomide and their last line of therapy. The report details clinical efficacy (PFS, ORR, OS) and safety findings, including common adverse events like thrombocytopenia and neutropenia, comparing melflufen plus dexamethasone to pomalidomide plus dexamethasone. The assessment highlights a positive benefit-risk balance for an earlier line of treatment, despite hematological safety concerns, particularly considering the impact of prior autologous stem cell transplantation (ASCT) status.
Key findings — cited to the page
| grade 3 or 4 infections (pomalidomide + dexamethasone arm) | 13.2 % | OP-103 · p.70 |
| grade 3/4 TEAEs (overall population) | 89.9 % | melflufen · p.76 |
| grade 3/4 TEAEs (melflufen + dexamethasone arm) | 10 % difference | OP-103 · p.70 |
| grade 3 or 4 infections (melflufen + dexamethasone arm) | 21.5 % | OP-103 · p.70 |
| severe (Grade 3) bleeding events (AESI) with thrombocytopenia | 0.9 % | melflufen · p.76 |
| patients had infection and concomitant Grade 3 or 4 neutropenia | 12.7 % | melflufen · p.76 |
| grade 3 or 4 thrombocytopenia and concomitant haemorrhage | 14.5 % | melflufen · p.69 |
| AESI of infection and concomitant Grade 3 or 4 neutropenia | 21.2 % | melflufen · p.69 |
| grade 5 TEAEs (melflufen + dexamethasone arm) | 11.8 % | OP-103 · p.70 |
| grade 5 TEAEs (pomalidomide + dexamethasone arm) | 13.0 % | OP-103 · p.70 |
| severe (Grade 3) bleeding events (AESI) with thrombocytopenia | 2 patients | melflufen · p.76 |
| grade 3/4 events (melflufen vs comparator) | 89.9 % | melflufen · p.76 |
Source: Oncopeptides ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →