| Generic name | Melphalan flufenamide |
| Brand name(s), other common name(s) | Pepaxto® |
| Drug type | Alkylating drug |
| How the drug is given | Injection, for intravenous use |
Indications and Usage
Melphalan flufenamide is given in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.
This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
Limitations of Use: Pepaxto is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.
Side effects needing medical attention
Most common adverse reactions are fatigue, nausea, diarrhea, fever and respiratory tract infection.
Most common laboratory abnormalities are leukocytes decrease, platelets decrease, lymphocytes decrease, neutrophils decrease, hemoglobin decrease and creatinine increase.
For information on how to manage the costs of drug therapy, please see Financial Support, or to speak with an Information Specialist, call (800) 955-4572.