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Liposomal irinotecan
Liposomal irinotecan is a Topoisomerase I inhibitor Small molecule drug developed by St. Jude Children's Research Hospital. It is currently in Phase 3 development for Metastatic pancreatic cancer (in combination with fluorouracil and leucovorin), Pediatric solid tumors. Also known as: (Onivyde®), Nal-IRI, nal-IRI, Irinotecan Liposome.
Liposomal irinotecan is a nanoparticle formulation of the topoisomerase I inhibitor irinotecan that delivers the chemotherapy agent to tumor cells while reducing systemic toxicity.
Liposomal irinotecan is a small molecule that inhibits DNA topoisomerase 1, a key enzyme involved in DNA replication. It is being studied in clinical trials for various soft tissue sarcoma conditions, including adult and childhood rhabdomyosarcoma and metastatic soft tissue sarcoma.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Liposomal irinotecan |
|---|---|
| Also known as | (Onivyde®), Nal-IRI, nal-IRI, Irinotecan Liposome, MM-398 |
| Sponsor | St. Jude Children's Research Hospital |
| Drug class | Topoisomerase I inhibitor |
| Target | Topoisomerase I |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Irinotecan inhibits topoisomerase I, an enzyme essential for DNA replication and transcription, causing DNA damage and cell death in rapidly dividing cancer cells. The liposomal encapsulation allows preferential accumulation in tumor tissue through enhanced permeability and retention (EPR), potentially improving the therapeutic index by reducing exposure to healthy tissues.
Approved indications
- Metastatic pancreatic cancer (in combination with fluorouracil and leucovorin)
- Pediatric solid tumors
Common side effects
- Neutropenia
- Diarrhea
- Nausea and vomiting
- Anemia
- Fatigue
Key clinical trials
- Studying Chemotherapy With or Without Panitumumab for Unresectable, Locally Advanced, or Metastatic Pancreatic Cancer Without KRAS Mutations (PHASE3)
- Oxaliplatin Combined With Irinotecan Liposome Injection II Through Hepatic Artery Infusion (HAIC) Followed by 5-FU/LV (HAIC) or Tegorgor Oral Combination Therapy for Hepatic Metastasis of Pancreatic Cancer (PHASE2)
- A Study to Evaluate the Effectiveness and Safety of Setidegrasib, Given With Either mFOLFIRINOX or NALIRIFOX Chemotherapies, in People With Pancreatic Cancer (PHASE3)
- Comparing Two Treatment Combinations, Gemcitabine and Nab-Paclitaxel With 5-Fluorouracil, Leucovorin, and Liposomal Irinotecan for Older Patients With Pancreatic Cancer That Has Spread (PHASE2)
- Apatinib Combined With Liposomal Irinotecan for Refractory or Metastatic Osteosarcoma (PHASE1, PHASE2)
- Monthly Alternating NALIRIFOX and GnP in the First-Line Setting for Metastatic Pancreatic Ductal Adenocarcinoma (PHASE2)
- SCRT-NALIRIXELOX+Sintilimab as TNT for High-Risk LARC (NA)
- Metastatic Ewing's Trial Testing Schedule Enhancement to Improve Outcomes (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Liposomal irinotecan CI brief — competitive landscape report
- Liposomal irinotecan updates RSS · CI watch RSS
- St. Jude Children's Research Hospital portfolio CI
Frequently asked questions about Liposomal irinotecan
What is Liposomal irinotecan?
How does Liposomal irinotecan work?
What is Liposomal irinotecan used for?
Who makes Liposomal irinotecan?
Is Liposomal irinotecan also known as anything else?
What drug class is Liposomal irinotecan in?
What development phase is Liposomal irinotecan in?
What are the side effects of Liposomal irinotecan?
What does Liposomal irinotecan target?
Related
- Drug class: All Topoisomerase I inhibitor drugs
- Target: All drugs targeting Topoisomerase I
- Manufacturer: St. Jude Children's Research Hospital — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Metastatic pancreatic cancer (in combination with fluorouracil and leucovorin)
- Indication: Drugs for Pediatric solid tumors
- Also known as: (Onivyde®), Nal-IRI, nal-IRI, Irinotecan Liposome, MM-398
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing