HEPZATO (melphalan hydrochloride)
HEPZATO is indicated for the treatment of Multiple Myeloma.
Details
- Status
- Prescription
- First Approved
- 2023-08-14
- Patent Cliff
- 2034
- Routes
- INTRA-ARTERIAL
- Dosage Forms
- POWDER
HEPZATO Approval History
What HEPZATO Treats
1 indicationsHEPZATO is approved for 1 conditions since its original approval in 2023. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Multiple Myeloma
HEPZATO Boxed Warning
WARNING Melphalan should be administered under the supervision of a qualified physician experienced in the use of cancer chemotherapeutic agents. Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) to oral melphalan have shown more myelosuppression with the IV formulation. Hypersensitivity reactions, including anaphylaxis, have occurred in approximately 2% of patients who received the IV formulation. Melphalan is leukemogeni...
WARNING Melphalan should be administered under the supervision of a qualified physician experienced in the use of cancer chemotherapeutic agents. Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) to oral melphalan have shown more myelosuppression with the IV formulation. Hypersensitivity reactions, including anaphylaxis, have occurred in approximately 2% of patients who received the IV formulation. Melphalan is leukemogenic in humans. Melphalan produces chromosomal aberrations in vitro and in viv o and, therefore, should be considered potentially mutagenic in humans.
HEPZATO Target & Pathway
ProTarget
HEPZATO Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in HEPZATO's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications HEPZATO treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to HEPZATO
3 of 20FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
8 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03192397 results posted | I 44417 NCI-2017-01069, I 44417 | Ph 1, Ph 2 | active not recruiting | Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant |
| NCT03126916 | ANBL1531 NCI-2016-01734, ANBL1531 | Ph 3 | recruiting | Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) |
| NCT03417284 results posted | 2017-0399 NCI-2018-00906, 2017-0399 | Ph 1, Ph 2 | completed | Melphalan Hydrochloride in Treating Participants With Newly-Diagnosed Multiple Myeloma Undergoing Donor Stem Cell Transplantation |
| NCT03128359 results posted | 16419 NCI-2017-00480, 16419 | Ph 2 | completed | High Dose Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil in Preventing Graft Versus Host Disease in Patients With Hematological Malignancies Undergoing Myeloablative or Reduced Intensity Donor Stem Cell Transplant |
| NCT02756572 results posted | 9567 NCI-2016-00477, 9567 | Ph 2 | completed | Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms |
| NCT03328936 | OSU-17082 NCI-2017-01702, P30CA016058 | Ph 2 | withdrawn | Study of Personalized Melphalan Dosing in the Setting of Autologous Transplant |
| NCT01688401 | J11164 NA_00069122 | Ph 1 | completed | Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG). |
| NCT01293539 results posted | J1071 NA_00040637 | Ph 2 | terminated | Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
HEPZATO FDA Label Details
Indications & Usage
FDA Label (PDF)HEPZATO is indicated for the treatment of Multiple Myeloma.
WARNING Melphalan should be administered under the supervision of a qualified physician experienced in the use of cancer chemotherapeutic agents. Severe bone marrow suppression with resulting infection or bleeding may occur. Controlled trials comparing intravenous (IV) to oral melphalan have shown m...
HEPZATO Patents & Exclusivity
Patents (10 active)
Exclusivity
Pro Intelligence Preview
Deep insights for HEPZATO
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2034
- • 11 active patents
Trial Analysis
- • Clinical trial tracking
- • Development stage analysis
Competitive Landscape
- • 20 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.