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FDA Approval and HORIZON Trial Efficacy Data
August 2021: Melphalan flufenamide (Pepaxto, Oncopeptides AB) in combination with dexamethasone has been granted accelerated approval by the Food and Drug Administration 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.
HORIZON (NCT02963493), a multicenter, single-arm trial, was used to assess efficacy. Patients had to have relapsed refractory multiple myeloma to be eligible. Patients received 40 mg of melphalan flufenamide intravenously on day 1 and 40 mg of dexamethasone orally on days 1, 8, 15, and 22 of each 28-day cycle until disease progression or intolerable toxicity.
Efficacy was assessed in a subset of 97 patients who had failed at least one proteasome inhibitor, one immunomodulatory drug, and a CD38-directed antibody and had had four or more prior lines of therapy. The overall response rate (ORR) and duration of response (DOR) were the primary efficacy outcomes measured by investigators using the International Myeloma Working Group (IMWG) Criteria. The ORR was 23.7 percent (95 percent confidence interval: 15.7 to 33.4), with a median DOR of 4.2 months (95 percent CI: 3.2, 7.6).
Safety Profile, Dosage, and Usage Guidelines
The 157 participants enrolled in HORIZON were examined for safety. Fatigue, nausea, diarrhoea, pyrexia, and respiratory tract infection are the most common side effects (> 20%). Decreased leukocytes, platelets, lymphocytes, neutrophils, and haemoglobin, as well as increased creatinine, are the most prevalent laboratory abnormalities (50 percent).
Melphalan flufenamide’s safety and efficacy as a conditioning regimen in transplant recipients has yet to be determined. Outside of controlled clinical trials, melphalan flufenamide is not indicated and is not recommended for use as a conditioning regimen for transplant, according to the USPI.
The recommended dose of melphalan flufenamide in conjunction with dexamethasone is 40 mg intravenously over 30 minutes on day 1 of each 28-day therapy cycle.
Reference:
Take second opinion on multiple myeloma treatment
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About Susan Hau
Susan Hau is a distinguished researcher in the field of cancer cell therapy, with a particular focus on T cell-based approaches and cancer vaccines. Her work spans several innovative treatment modalities, including CAR T-cell therapy, TIL (Tumor-Infiltrating Lymphocyte) therapy, and NK (Natural Killer) cell therapy. Hau's expertise lies in cancer cell biolo…
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This article is for educational purposes only and should not replace medical advice, diagnosis, or treatment from a qualified oncology specialist. Every patient's case is different. Treatment decisions should always be made after a review of complete medical records by the treating medical team.
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