Oncopeptides — Pepaxti EMA EPAR public assessment report (EMEA/H/C/005681, 2022)
Oncopeptides · 2022 · EMA EPAR public assessment report
Report summary
This EMA public assessment report details the regulatory evaluation of Pepaxti (melphalan flufenamide) for triple-class refractory multiple myeloma. Preclinical studies demonstrated melflufen's superior cytotoxicity and tumor inhibition compared to melphalan, acting as a prodrug. Clinical trials (HORIZON and OCEAN) showed efficacy, with an objective response rate of 29.3% and a median progression-free survival of 6.83 months in the OCEAN study. However, the OCEAN study also revealed a detrimental overall survival for melflufen compared to pomalidomide, particularly in patients with early relapse post-ASCT. Safety concerns include high rates of hematologic toxicities (thrombocytopenia, neutropenia) and potential secondary primary malignancies. The report concludes with a restricted indication for Pepaxti due to these safety and efficacy concerns, especially for patients with early relapse after ASCT.
Key findings — cited to the page
| patients with Grade 3 melflufen-related SAE | 11 % | Study OP-106 · p.110 |
| patients with Grade 4 melflufen-related AEs | 65.6 % | Study OP-106 · p.108 |
| patients with Grade 3 dexamethasone-related TEAEs | 31.8 % | Study OP-106 · p.108 |
| patients with Grade 4 dexamethasone-related TEAEs | 9.6 % | Study OP-106 · p.108 |
| patients with Grade 3 or 4 treatment-related AEs (any study drug) | 89.8 % | Study OP-106 · p.108 |
| patients with Grade 3 melflufen-related AEs | 24.2 % | Study OP-106 · p.108 |
| patients with Grade 3 or 4 TEAEs | 93.6 % | Study OP-106 · p.104 |
| patients with Grade 3 or 4 neutropenia | 52.9 % | Study OP-106 · p.107 |
| patients with Grade 3 or 4 thrombocytopenia | 56.7 % | Study OP-106 · p.107 |
| patients with melflufen-related Grade 3 or 4 TEAEs | 89.8 % | Study OP-106 · p.104 |
| patients with Grade 3 or 4 anaemia | 42.7 % | Study OP-106 · p.107 |
| patients with Grade 4 melflufen-related SAE | 8 % | Study OP-106 · p.111 |
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 →