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 has been granted under accelerated approval based upon the HORIZON trial. PEPAXTO is the first anticancer peptide-drug conjugate approved in multiple myeloma.
About PEPAXTO
PEPAXTO(melphalan flufenamide)is the first anticancer peptide-drug conjugate for patients with triple-class refractory Multiple Myeloma who have received at least four prior lines of therapy. PEPAXTO uses innovative technology that links a peptide carrier to a cytotoxic agent, resulting in a lipophilic compound. Due to its lipophilicity, PEPAXTO is distributed into cells. PEPAXTO is designed to leverage aminopeptidases, which are overexpressed in multiple myeloma cells and cause the release of the cytotoxic agents. PEPAXTO is administered as a once monthly thirty-minute infusion.
INDICATION
PEPAXTO is indicated 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 a confirmatory trial.
Limitation of Use
PEPAXTO is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.
IMPORTANT SAFETY INFORMATION
PEPAXTO is contraindicated in patients with a history of serious allergic reaction to melphalan flufenamide or melphalan.
PEPAXTO may cause thrombocytopenia, which may lead to hemorrhage.Monitor platelets at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if the platelet count is less than 50 x 109/L. Withhold PEPAXTO until platelet count is 50 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Adjust dose and/or dose schedule based on signs and symptoms of bleeding.
PEPAXTO may cause neutropenia, which may lead to infection. Monitor neutrophil counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Do not administer PEPAXTO if absolute neutrophil count is less than 1 x 109/L. Withhold PEPAXTO until absolute neutrophil count is 1 x 109/L or greater and resume at same or reduced dose based on duration of interruption. Consider leukocyte growth factor as clinically appropriate.
PEPAXTO may cause anemia. Monitor red blood cell counts at baseline, during treatment, and as clinically indicated. Monitor more frequently during the first 2 months of treatment with PEPAXTO. Treat anemia as clinically indicated. Dosage modification and dose delay of PEPAXTO may be required to allow for recovery of red blood cells.
Patients taking PEPAXTO experienced infections, including fatal infections. Consider antimicrobials as clinically appropriate.
Nonclinical safety studies with melphalan flufenamide at dosages exceeding the recommended dose for PEPAXTO were associated with mortality. The safety and effic |
