HEPZATO (melphalan hydrochloride) · DELCATH SYSTEMS INC
HEPZATO for injection, as a component of the HEPZATO KIT, is indicated as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.
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.
HEPZATO Boxed Warning
PERI-PROCEDURAL COMPLICATIONS, MYELOSUPPRESSION Severe peri-procedural complications including hemorrhage, hepatocellular injury, and thromboembolic events may occur via hepatic intra-arterial administration of HEPZATO. Assess patients for these adverse reactions during and for at least 72 hours following administration of HEPZATO [see Warnings and Precautions ( 5.1 )]. HEPZATO is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the HEPZATO KIT R...
WARNING: PERI-PROCEDURAL COMPLICATIONS, MYELOSUPPRESSION Severe peri-procedural complications including hemorrhage, hepatocellular injury, and thromboembolic events may occur via hepatic intra-arterial administration of HEPZATO. Assess patients for these adverse reactions during and for at least 72 hours following administration of HEPZATO [see Warnings and Precautions ( 5.1 )]. HEPZATO is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the HEPZATO KIT REMS [see Warnings and Precautions ( 5.2 )]. Myelosuppression with resulting severe infection, bleeding, or symptomatic anemia may occur with HEPZATO. Monitor hematologic laboratory parameters and delay additional cycles of HEPZATO therapy until blood counts have improved. [see Warnings and Precautions ( 5.1 )] WARNING: SEVERE PERI-PROCEDURAL COMPLICATIONS, MYELOSUPPRESSION See full prescribing information for complete boxed warning. Severe peri-procedural complications including hemorrhage, hepatocellular injury, and thromboembolic events may occur with intra-hepatic administration of HEPZATO. Assess patients for these adverse reactions during and for 72 hours following administration of HEPZATO. ( 5.1 ) HEPZATO is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the HEPZATO KIT REMS. ( 5.2 ) Myelosuppression with resulting severe infection, bleeding, or symptomatic anemia may occur with HEPZATO. Monitor hematologic laboratory parameters and delay additional cycles of HEPZATO therapy until blood counts have improved.( 5.3 )
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 for injection, as a component of the HEPZATO KIT, is indicated as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation. HEPZATO is an alkylating drug indicated as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepati...
WARNING: PERI-PROCEDURAL COMPLICATIONS, MYELOSUPPRESSION Severe peri-procedural complications including hemorrhage, hepatocellular injury, and thromboembolic events may occur via hepatic intra-arterial administration of HEPZATO. Assess patients for these adverse reactions during and for at least 72 ...
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.