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Melphalan (including hepatic delivery system)

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.

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.

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Melphalan
PubChem
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.
Brand / code
Alkeran / Evomela / Hepzato Kit
Medicare
Part B or Part D

Oral anticancer drug with an injectable equivalent used for the same indication, so Medicare covers the oral form under Part B (the oral anticancer drug benefit) as well as under Part D; the pharmacy bills whichever applies. High-dose IV melphalan for transplant conditioning is inpatient (Part A). The Hepzato Kit (hepatic delivery) is a hospital procedure with its own coding.

Commercial insurance
covered on label

Multi-source generic; covered under the medical benefit without prior authorisation in most plans, as part of standard regimens. Hepzato Kit requires prior authorisation.

Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

Not appraisedSearch NICE
Notes
Generic melphalan is standard for autologous transplant conditioning in myeloma and funded routinely. The Hepzato hepatic delivery system for uveal melanoma liver metastases is not UK-licensed.
NHS England
Routinely funded for the appraised indication (or via managed access)

Sources: NICE search: melphalan. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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Approvals

RegionYearIndication
US1964Multiple myeloma; non-resectable epithelial ovarian carcinoma
US2016High-dose conditioning before autologous HSCT in myeloma (Evomela)
US2023Uveal melanoma with unresectable hepatic metastases via hepatic delivery system (Hepzato Kit)

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
728 studies10 recruiting74 Phase 229 Phase 3
Search “Melphalan” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Melphalan (including hepatic delivery system)
intervention: Melphalan
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Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Melphalan" OR ABSTRACT:"Melphalan" OR TITLE:"including hepatic delivery system" OR ABSTRACT:"including hepatic delivery system" OR TITLE:"Alkeran" OR ABSTRACT:"Alkeran" OR TITLE:"Evomela" OR ABSTRACT:"Evomela" OR TITLE:"Hepzato Kit" OR ABSTRACT:"Hepzato Kit") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Melphalan (including hepatic delivery system), not a curated reading list.

Connected

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