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Drug

Melphalan hydrochloride

Approved · EMA
Late-stage development
Also known as Alkeran, Evomela, Melphalan, melphalan (as hydrochloride)+1 more

Alkeran, Evomela, Melphalan, melphalan (as hydrochloride), melphalan HCl.

RxNorm235857
2
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase Ib/2 Trial of Fludarabine/Melphalan/Total Body Irradiation With Post Transplant Cyclophosphamide as Graft Versus Host Disease Prophylaxis in Matched-Related and Matched-Unrelated Allogeneic Hematopoietic Cell Transplantation

Clinical trial2026-04-14Results posted · ClinicalTrials.gov

A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)

Clinical trial2026-02-27Results expected Q3 2030 · ClinicalTrials.gov

Prospective Phase I/II Trial to Jointly Optimize the Administration Schedule(s) and Dose(s) of Melphalan for Injection (Evomela) as a Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation in Patients With Newly Diagnosed Multiple Myeloma

Clinical trial2025-05-01Results posted · ClinicalTrials.gov

A Pilot Study of Post-transplant High Dose Cyclophosphamide (PTCY) as Part of Graft-Versus-Host Disease (GVHD) Prophylaxis in T-Cell Replete HLA-Mismatched Unrelated Donor (MMUD) Ablative and Reduced Intensity Hematopoietic Cell Transplantation (HCT) for Hematological Malignancies

Clinical trial2024-01-09Results posted · ClinicalTrials.gov

A Feasibility Study of "Early" Allogeneic Hematopoietic Cell Transplantation for Relapsed or Refractory High-Grade Myeloid Neoplasms

Clinical trial2021-11-08Results posted · ClinicalTrials.gov

Randomized Study of Personalized Melphalan Dosing in the Setting of Autologous Transplant

Clinical trial2021-03-31Withdrawn · ClinicalTrials.gov

Approval: Phelinun (EMA)

Regulatory2020-11-16EMA

Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).

Clinical trial2018-11-26Completed · ClinicalTrials.gov

A Phase II Study of Melphalan HCl for Injection (Propylene Glycol-free), Combined With Carmustine, Etoposide, and Cytarabine (BEAM Regimen) for Myeloablative Conditioning in Lymphoma Patients Undergoing Autologous Stem Cell Transplantation

Clinical trial2018-02-13Results posted · ClinicalTrials.gov

Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Clinical trial2016-08-01Terminated · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Melphalan hydrochloride
Aliases & brands
AlkeranEvomelaMelphalanmelphalan (as hydrochloride)melphalan HCl
RxNorm CUI
235857
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2020-11-16
Latest approval
2020-11-16
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2020-11-16Approval
Approval: Phelinun (EMA)
Indication: High-dose of Phelinun used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: multiple myeloma,… Show full indication

High-dose of Phelinun used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: multiple myeloma, malignant lymphoma (Hodgkin, non-Hodgkin lymphoma), acute lymphoblastic and myeloblastic leukemia, childhood neuroblastoma, ovarian cancer, mammary adenocarcinoma. Phelinun in combination with other cytotoxic medicinal products is indicated as reduced intensity conditioning (RIC) treatment prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in malignant haematological diseases in adults. Phelinun in combination with other cytotoxic medicinal products is indicated as conditioning regimen prior to allogeneic haematopoietic stem cell transplantation in haematological diseases in the paediatric population as: Myeloablative conditioning (MAC) treatment in case of malignant haematological diseases RIC treatment in case of non-malignant haematological diseases.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

9 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
9
registered trials across all phases
LATEST COMPLETION 2024
2
Active studies
1
Recruiting
1
Late-stage (III+)
5
Completed
2
Discontinued
PHASE DISTRIBUTIONn = 9
Phase 11Phase 1 / 22Phase 25Phase 31

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)

RecruitingPH IIIChildren's Oncology Groupn=7502030

Recent completions

Trials that read out recently, adding to the completed evidence base.

Prospective Phase I/II Trial to Jointly Optimize the Administration Schedule(s) and Dose(s) of Melphalan for Injection (Evomela) as a Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation in Patients With Newly Diagnosed Multiple Myeloma

CompletedPH I/IIM.D. Anderson Cancer Centern=622024

A Pilot Study of Post-transplant High Dose Cyclophosphamide (PTCY) as Part of Graft-Versus-Host Disease (GVHD) Prophylaxis in T-Cell Replete HLA-Mismatched Unrelated Donor (MMUD) Ablative and Reduced Intensity Hematopoietic Cell Transplantation (HCT) for Hematological Malignancies

CompletedPH IICity of Hope Medical Centern=382021

A Feasibility Study of "Early" Allogeneic Hematopoietic Cell Transplantation for Relapsed or Refractory High-Grade Myeloid Neoplasms

CompletedPH IIUniversity of Washingtonn=302020

Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).

CompletedPH ISidney Kimmel Comprehensive Cancer Center at Johns Hopkinsn=32018

A Phase II Study of Melphalan HCl for Injection (Propylene Glycol-free), Combined With Carmustine, Etoposide, and Cytarabine (BEAM Regimen) for Myeloablative Conditioning in Lymphoma Patients Undergoing Autologous Stem Cell Transplantation

CompletedPH IIWashington University School of Medicinen=502017
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.