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                Resources for Information | Approved Drugs

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                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
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          FDA D.I.S.C.O. Burst: Approval of Pepaxto (melphalan flufenamide) in combination with dexamethasone for adult patients with relapsed or refractory multiple myeloma who have received at least four lines of prior therapyResources for Information | Approved Drugs FDA D.I.S.C.O. Burst: Approval of Pepaxto (melphalan flufenamide) in combination with dexamethasone for adult patients with relapsed or refractory multiple myeloma who have received at least four lines of prior therapy
  
  	
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Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we have another quick update on a recent FDA cancer drug approval.

On February 26, 2021, the FDA granted approval to melphalan flufenamide (brand name Pepaxto) in combination with dexamethasone for 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 CD-38 directed monoclonal antibody.

Efficacy was evaluated in HORIZON, a multicenter, single-arm trial. Eligible patients were required to have relapsed refractory multiple myeloma. Patients received melphalan flufenamide on day 1 and dexamethasone orally on day 1, 8, 15 and 22 of each 28-day cycle until disease progression or unacceptable toxicity.

Efficacy was evaluated in a subpopulation of 97 patients who received four or more prior lines of therapy and were refractory to at least one proteasome inhibitor, one immunomodulatory agent, and a CD38-directed antibody. The main efficacy outcome measure was overall response rate and duration of response assessed by investigators according to the International Myeloma Working Group Criteria. The overall response rate was 23.7% and median duration of response was 4.2 months.

The safety and efficacy of melphalan flufenamide has not been established for use as a conditioning regimen in patients receiving transplant. The prescribing information includes a Limitations of Use statement that melphalan flufenamide is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.

Full prescribing information for this approval can be found on the web at www.fda.gov/drugsatFDA.

Health care professionals should report serious adverse events to FDA’s MedWatch Reporting System at www.fda.gov/medwatch.

Follow the Division of Drug Information on Twitter @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology. Send your feedback via email to FDAOncology@fda.hhs.gov. Thanks again for tuning into the DISCO Burst Edition.

                                                                                  Content current as of:
      03/16/2021
              
              
Regulated Product(s)
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						  OncologyResources for Information | Approved Drugs

          
                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
          Ongoing | Cancer Accelerated Approvals
          Verified Clinical Benefit | Cancer Accelerated Approvals
          Withdrawn | Cancer Accelerated Approvals
          Other | Cancer Accelerated Approvals


                        Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description
 
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