Melphalanflufenamid (1) – Pepaxti®
Multiple myeloma (MM) after at least 3 previous therapies, combination with dexamethasone)
Characteristics
| Start date | 01.10.2022 – Marketing authorisation: 17.08.2022 |
|---|---|
| Resolution | 16.03.2023 |
| INN | Melphalanflufenamid |
| Brand name | Pepaxti® |
| Pharm. company | Oncopeptides AB |
| G-BA Procedure ID | D-868 |
| ATC code | L01AA10 Nitrogen mustard analogues (L01AA) |
| ICD-10 codes (AIS) | C90.00Multiple myeloma with failed remission, C90.01Multiple myeloma in remission |
| Alpha-ID codes (AIS) | I21328Multiple myeloma, I31059Multiple myeloma in complete remission |
| Therapeutic area | Oncological diseases Multiple myeloma (MM) |
| Reason for procedure | Initial assessment |
| Specialty | ACT change |
| Therapeutic indication of the resolution |
|---|
|
Pepaxti is indicated in combination with dexamethasone for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapy, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one CD38 monoclonal antibody, and who have demonstrated disease progression during or after the last line of therapy. In patients with previous autologous stem cell transplantation, the time to progression after transplantation should be at least 3 years. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Adults with multiple myeloma who have received at least three prior lines of therapy, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38 monoclonal antibody, and who have demonstrated disease progression during or after the last therapy; time to progression at least three years for those with prior autologous stem cell transplantation | Patient-individual therapy with selection of: - Bortezomib monotherapy - Bortezomib + pegylated liposomal doxorubicin - Bortezomib + dexamethasone - Carfilzomib + lenalidomide and dexamethasone - Carfilzomib + dexamethasone - Daratumumab + lenalidomide + dexamethasone - Daratumumab + bortezomib + dexamethasone - Daratumumab monotherapy (only for people with disease progression on last therapy) - Daratumumab + pomalidomide + dexamethasone - Elotuzumab + lenalidomide + dexamethasone - Elotuzumab + pomalidomide + dexamethasone (only for people with disease progression on last therapy) Isatuximab + pomalidomide + dexamethasone (only for people with disease progression on last therapy) - Ixazomib + lenalidomide + dexamethasone - Lenalidomide + dexamethasone - Panobinostat + bortezomib and dexamethasone - Pomalidomide + bortezomib and dexamethasone - Pomalidomide + dexamethasone (only for those with disease progression on last therapy) - Cyclophosphamide (in combination with other antineoplastic drugs) - Melphalan - Doxorubicin - Carmustine (in combination with other cytostatic drugs and an adrenocortical hormone, especially prednisone) - vincristine - Dexamethasone - Prednisolone - prednisone - Best Supportive Care taking into account previous therapies and the degree and duration of response. |
Studies and Results
|
No. of studies
(best subpopulation) |
1 (OCEAN) 0 (Data not accepted) |
|---|---|
|
Study design
(best subpopulation) |
Data not accepted (Dossier: H2H vs. ACT) |
| ACT change | 27.09.2022 – Stellungnahme der Fachgesellschaften, Neue G-BA Beschlüsse |
- Clinical trials
- In the randomised, open-label, controlled Phase III OCEAN trial, treatment with melphalan flufenamide in combination with dexamethasone was compared with the combination therapy of pomalidomide and dexamethasone (Pd).
- The single-arm, open-label Phase II HORIZON trial, which investigated treatment with melphalan flufenamide in combination with dexamethasone, enrolled patients with relapsed, refractory multiple myeloma who had received at least two prior lines of treatment, including an immunomodulatory agent and a proteasome inhibitor.
Adults with multiple myeloma who have previously received at least three lines of treatment, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one monoclonal CD38 antibody, and who have shown disease progression during or after their most recent treatment; time to progression of at least three years in patients who have previously undergone an autologous stem cell transplant
- An additional benefit is not proven.
- There are therefore no suitable data available to demonstrate any additional benefit of melphalan flufenamide in combination with dexamethasone compared with the appropriate comparator therapy.
- Overall assessment
- As there are therefore no suitable data to provide proof of an additional benefit of melphalan flufenamide in combination with dexamethasone compared with the appropriate comparator therapy, an additional benefit of melphalan flufenamide in combination with dexamethasone is not proven.
Courtesy translation only, please refer to the German original.
Associated procedures
<< List of all resolutions