{"entity":{"id":"melphalan","kind":"drug","name":"Melphalan (including hepatic delivery system)","aka":[],"tldr":"The myeloma chemotherapy that is also the standard high-dose conditioning before autologous transplant, and, delivered directly into the liver's blood supply or the eye, treats uveal melanoma metastases and retinoblastoma.","summary":"Approved 1964 (oral, myeloma; melphalan-prednisone was the standard for decades). High-dose melphalan 200 mg/m² with autologous stem-cell rescue remains standard consolidation in myeloma (IFM 2009, DETERMINATION). Hepzato Kit (2023): percutaneous hepatic perfusion for uveal melanoma liver metastases (FOCUS trial, 36% response). Intra-arterial and intravitreal melphalan are standard in retinoblastoma. Melflufen is a peptide-conjugated prodrug.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Melphalan","links":[{"label":"Label: Hepzato Kit (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Hepzato"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=melphalan"}],"tags":["gap-fill","generic"],"related":[],"cancers":["multiple-myeloma","uveal-melanoma","retinoblastoma","ovarian","myeloma-transplant-eligible"],"sections":[],"technologies":["cytotoxic-chemotherapy","autologous-stem-cell-transplant","percutaneous-hepatic-perfusion","isolated-limb-perfusion"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05022901","nct06607458"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Alkeran / Evomela / Hepzato Kit","modality":"Alkylating agent (nitrogen mustard)","mechanism":"Bifunctional alkylator producing DNA interstrand cross-links; active in myeloma at conventional dose and as high-dose conditioning; regional delivery for eye melanoma liver metastases and retinoblastoma.","approvals":[{"region":"US","year":1964,"indication":"Multiple myeloma; non-resectable epithelial ovarian carcinoma"},{"region":"US","year":2016,"indication":"High-dose conditioning before autologous HSCT in myeloma (Evomela)"},{"region":"US","year":2023,"indication":"Uveal melanoma with unresectable hepatic metastases via hepatic delivery system (Hepzato Kit)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/melphalan/","neighbours":{"cancer":[{"id":"dlbcl","kind":"cancer","name":"Diffuse large B-cell lymphoma","route":"/cancers/dlbcl/"},{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"multiple-myeloma","kind":"cancer","name":"Multiple myeloma","route":"/cancers/multiple-myeloma/"},{"id":"myeloma-transplant-eligible","kind":"cancer","name":"Newly diagnosed multiple myeloma, transplant-eligible","route":"/cancers/myeloma-transplant-eligible/"},{"id":"myeloma-transplant-ineligible","kind":"cancer","name":"Newly diagnosed multiple myeloma, transplant-ineligible","route":"/cancers/myeloma-transplant-ineligible/"},{"id":"ovarian","kind":"cancer","name":"Ovarian cancer","route":"/cancers/ovarian/"},{"id":"peripheral-t-cell-lymphoma","kind":"cancer","name":"Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma)","route":"/cancers/peripheral-t-cell-lymphoma/"},{"id":"plasma-cell-leukaemia","kind":"cancer","name":"Plasma cell leukaemia","route":"/cancers/plasma-cell-leukaemia/"},{"id":"relapsed-refractory-hodgkin-lymphoma","kind":"cancer","name":"Relapsed and refractory classical Hodgkin lymphoma","route":"/cancers/relapsed-refractory-hodgkin-lymphoma/"},{"id":"retinoblastoma","kind":"cancer","name":"Retinoblastoma","route":"/cancers/retinoblastoma/"},{"id":"uveal-melanoma","kind":"cancer","name":"Uveal melanoma","route":"/cancers/uveal-melanoma/"}],"technology":[{"id":"autologous-stem-cell-transplant","kind":"technology","name":"Autologous stem cell transplant (high-dose therapy)","route":"/technologies/autologous-stem-cell-transplant/"},{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"},{"id":"isolated-limb-perfusion","kind":"technology","name":"Isolated limb perfusion and infusion","route":"/technologies/isolated-limb-perfusion/"},{"id":"oral-cryotherapy-mucositis","kind":"technology","name":"Oral cryotherapy (ice chips) to prevent mucositis","route":"/technologies/oral-cryotherapy-mucositis/"},{"id":"percutaneous-hepatic-perfusion","kind":"technology","name":"Percutaneous hepatic perfusion (chemosaturation)","route":"/technologies/percutaneous-hepatic-perfusion/"}],"trial":[{"id":"nct06679829","kind":"trial","name":"A Study of Melphalan With or Without Siltuximab in People With Multiple Myeloma Having an Autologous Stem Cell Transplant","route":"/trials/nct06679829/"},{"id":"nct05022901","kind":"trial","name":"An Open-Label Expanded Access Study of the Melphalan/Hepatic Delivery System (HDS) in Patients With Hepatic Dominant Ocular Melanoma","route":"/trials/nct05022901/"},{"id":"bmt-ctn-0803","kind":"trial","name":"BMT CTN 0803","route":"/trials/bmt-ctn-0803/"},{"id":"determination","kind":"trial","name":"DETERMINATION","route":"/trials/determination/"},{"id":"nct06607458","kind":"trial","name":"Evaluation of the Safety and Efficacy of Treatment w/High Dose Melphalan Given Directly Into the Liver Followed by Treatment w/Approved Cancer Treatme","route":"/trials/nct06607458/"},{"id":"nct02678572","kind":"trial","name":"FOCUS","route":"/trials/nct02678572/"},{"id":"nct04221035","kind":"trial","name":"High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)","route":"/trials/nct04221035/"},{"id":"nct02506959","kind":"trial","name":"Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan Before Stem Cell Transplant in Treating Patients With Refractory or Relapsed Multiple Myeloma","route":"/trials/nct02506959/"}],"term":[{"id":"alkylating-agent","kind":"term","name":"Alkylating agents and antimetabolites","route":"/terms/alkylating-agent/"},{"id":"rejuv-second-alkylating-agents-and-myeloid-neoplasms","kind":"term","name":"Alkylating agents and therapy-related myeloid neoplasms","route":"/terms/rejuv-second-alkylating-agents-and-myeloid-neoplasms/"},{"id":"autologous-transplant","kind":"term","name":"Autologous stem cell transplant (ASCT)","route":"/terms/autologous-transplant/"},{"id":"rejuv-second-choices-made-at-treatment","kind":"term","name":"Choices made at the time of treatment that change the second cancer risk","route":"/terms/rejuv-second-choices-made-at-treatment/"},{"id":"conditioning-regimen","kind":"term","name":"Conditioning regimen (myeloablative, reduced-intensity)","route":"/terms/conditioning-regimen/"},{"id":"lymphoma-tx-fertility-preservation","kind":"term","name":"Fertility before lymphoma treatment: what to ask for, and when","route":"/terms/lymphoma-tx-fertility-preservation/"},{"id":"rejuv-second-platinum-and-parp-inhibitors","kind":"term","name":"Platinum drugs and PARP inhibitors: the newer leukaemia risk","route":"/terms/rejuv-second-platinum-and-parp-inhibitors/"},{"id":"lymphoma-tx-transplant-role","kind":"term","name":"Stem cell transplant in lymphoma: what it is still for","route":"/terms/lymphoma-tx-transplant-role/"}],"drug":[{"id":"tasonermin","kind":"drug","name":"Tasonermin","route":"/drugs/tasonermin/"}],"company":[{"id":"acrotech-biopharma","kind":"company","name":"Acrotech Biopharma","route":"/companies/acrotech-biopharma/"}]}}