What is PEPAXTO®?
PEPAXTO, also known as melphalan flufenamide (also known as melflufen), is a first-in-class peptide-drug conjugate. PEPAXTO is manufactured by Oncopeptides.
As of 10/22/2021, Pepaxto was withdrawn from the US market, and is no longer available for new prescriptions. If you are currently on Pepaxto therapy, contact your treating physician to see if remaining on therapy is appropriate for you.
Important Notice
More Information
Uses
How is PEPAXTO used?
PEPAXTO, in combination with dexamethasone, is used for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) 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.What types of patients can benefit from PEPAXTO?
In a phase 2 clinical trial, PEPAXTO, in combination with dexamethasone, was shown to be effective in:- Patients who received at least four prior therapies
- Patients who were refractory to their previous treatment(s)
- Patients who previously received high-dose chemotherapy and stem cell transplant
How does PEPAXTO work?
PEPAXTO is a peptide conjugated cytotoxic drug. PEPAXTO is internalized by the myeloma cell. Once inside the cell, PEPAXTO releases the cytotoxic agent, leading to myeloma cell death.
Dosing and Administration
How is PEPAXTO administered?
PEPAXTO is given intravenously at a dose of 40 mg over 30 minutes once a month until disease progression.
Side Effects
What are PEPAXTO side effects?
The most common side effects seen in a clinical study with PEPAXTO included:- Fatigue
- Nausea
- Diarrhea
- Pyrexia
- Respiratory tract infection
- Low leukocyte counts (leukocytopenia)
- Low platelet counts (thrombocytopenia)
- Low lymphocyte counts (lymphopenia)
- Low neutrophil counts (neutropenia)
- Anemia (low hemoglobin)
- Increase in creatinine
- Low platelet counts (thrombocytopenia)
- Low neutrophil counts (neutropenia)
- Anemia (low hemoglobin)
- Infections
- Secondary malignancies
- Increased risk of death at doses higher than recommended
- Embryo-fetal toxicity
How are side effects of PEPAXTO managed?
Your doctor may prescribe an anti-nausea medication prior to and during the treatment with PEPAXTO.
Clinical Studies
What have PEPAXTO clinical trials shown?
The approval of PEPAXTO was based on results from the HORIZON study, a multicenter, single-arm trial to determine the efficacy of PEPAXTO in patients with RRMM. The study included 157 patients with relapsed or refractory myeloma of whom 97 were considered triple-class refractory (that is, patients who have received treatment with—and did not respond satisfactorily to, or progressed while on treatment with—the three main classes of drugs currently used to treat myeloma: proteasome inhibitors, immunomodulatory drugs, and anti-CD38 monoclonal antibodies). The response to PEPAXTO treatment was seen in 24% of the triple-class refractory patients and the median duration of response was 4.2 months. Furthermore, a subset of patients who had extramedullary disease (that is, myeloma cells present outside of the bone marrow) responded to the combination.How is PEPAXTO currently being studied in myeloma?
PEPAXTO is being evaluated in clinical trials in the U.S.- Phase 3 trial:
- PEPAXTO with dexamethasone or with Pomalyst (pomalidomide, BMS)-dexamethasone for patients with RRMM who are refractory to Revlimid (lenalidomide, BMS) (OCEAN trial; NCT03151811)
- Phase 1 and 2 trial:
- PEPAXTO with dexamethasone and Velcade (bortezomib, Takeda Oncology) or Darzalex (daratumumab, Janssen Biotech) for patients with RRMM (ANCHOR trial; NCT03481556)
Interested in learning more about PEPAXTO clinical trials? Talk to an MMRF patient navigator or use our clinical trials finder: FIND CLINICAL TRIALS
Created February 2021
