NCT03295396
- Objective
- Not yet disclosed
- Design
- Phase 2 study in glioma; specific design details not yet disclosed
- Participants
- Not yet disclosed
- Primary endpoint
- Not yet disclosed
- Results
- Results not yet reported; program terminated July 28, 2025
pharma · Acute Myeloid Leukemia · Narcolepsy · JAZZ
Jazz Pharmaceuticals Ireland
Jazz Pharmaceuticals Ireland Limited
Jazz Pharmaceuticals Ireland is a pharma organization headquartered in Dublin, IE. It trades on NYSE under ticker JAZZ. Primary therapeutic focus areas include Acute Myeloid Leukemia, Narcolepsy, Epilepsy, Acute Lymphobl
Waterloo Exchange, Waterloo Road, Dublin, 4, IE HQ
2003 Founded
3,631 Employees
Public company Type
JAZZ · NYSE Ticker
Company details
- Status
- Public
- HQ
- Waterloo Exchange, Waterloo Road, Dublin, 4, IE
- Founded
- 2003
- Employees
- 3,631
- Programs
- 208
- Drugs
- 91
- Patents
- 89
Dordaviprone (ONC201)
Phase 2 · small molecule · Glioma
Dordaviprone (ONC201, internal code ONC013) is a small-molecule oral therapeutic candidate developed by Jazz Pharmaceuticals Ireland Limited for glioma, currently in Phase 2 development. The program was terminated as of July 28, 2025. The mechanism of action and specific molecular target have not been disclosed. Jazz P
Internal code ONC013
At a glance
- Sponsor
- Jazz Pharmaceuticals Ireland Limited
- Phase
- Phase 2
- Modality
- small_molecule
- Indication
- Glioma
- Status
- terminated
- Trials
- 1
Executive summary
Dordaviprone (ONC201, internal code ONC013) is a small-molecule oral therapeutic candidate developed by Jazz Pharmaceuticals Ireland Limited for glioma, currently in Phase 2 development. The program was terminated as of July 28, 2025. The mechanism of action and specific molecular target have not been disclosed. Jazz Pharmaceuticals' strategy appears focused on oncology, with dordaviprone representing one of several investigational programs in the glioma space. The program's termination marks the end of clinical development for this indication, though the underlying compound dordaviprone hydrochloride carries the brand name MODEYSO and has achieved U.S. FDA approval under NDA219876, sponsored by Chimerix for an undisclosed indication. The termination of the glioma program does not affect the regulatory status of MODEYSO in other therapeutic contexts.
Why this program matters
Glioma represents a significant unmet medical need, particularly high-grade gliomas such as glioblastoma, which carry poor prognosis and limited treatment options despite standard-of-care chemotherapy and radiation. The termination of the dordaviprone glioma program reflects the competitive and challenging landscape in CNS oncology, where efficacy, blood-brain barrier penetration, and tolerability remain critical barriers. The competitive landscape includes approved agents such as AFINITOR (everolimus, Novartis), IMBRUVICA (ibrutinib, Janssen-Cilag), and INLYTA (axitinib, Pfizer), though these are primarily indicated for other malignancies and their glioma applications remain limited. The termination suggests either insufficient efficacy signals, safety concerns, or strategic portfolio prioritization by Jazz Pharmaceuticals. The market for glioma therapeutics remains substantial given the disease burden and mortality, but the failure of this program underscores the difficulty in translating preclinical promise into clinical benefit in this indication.
Drug intelligence
Dordaviprone hydrochloride is a small-molecule compound administered orally. The mechanism of action and molecular target have not been disclosed in available regulatory or clinical documentation. The compound is marketed as MODEYSO following FDA approval under NDA219876 by sponsor Chimerix, indicating prior development and regulatory success in a non-glioma indication. The glioma program (ONC013) represents a separate development pathway by Jazz Pharmaceuticals. Related investigational approaches in glioma include immunotherapeutic agents such as autologous glioma tumor cell lysates (SITOIGANAP), which was developed by Epitopoietic Research Corporation-Belgium but had its EMA application withdrawn.
- Modality: Small-molecule
- Route: Oral
- Approved formulation brand: MODEYSO (dordaviprone hydrochloride)
- Approved sponsor: Chimerix (NDA219876)
- Mechanism of action: Not yet disclosed
- Molecular target: Not yet disclosed
glioma
Also known as: glial neoplasm, glial tumor, glial tumour, neoplasm of neuroglia, neoplasm of the neuroglia, neuroglial neoplasm
Prevalence: Point prevalence: 1-5 / 10 000 (Europe) — source: Orphanet, validated.
Overview
A benign or malignant brain and spinal cord tumor that arises from glial cells (astrocytes, oligodendrocytes, ependymal cells). Tumors that arise from astrocytes are called astrocytic tumors or astrocytomas. Tumors that arise from oligodendrocytes are called oligodendroglial tumors. Tumors that arise from ependymal cells are called ependymomas.
Treatment landscape
ClinicalTrials.gov lists 517 registered studies for Glioma (AACT aggregate).
Phase breakdown: NA (265), PHASE1 (85), PHASE2 (82), PHASE1/PHASE2 (33), EARLY_PHASE1 (29), PHASE3 (13), PHASE2/PHASE3 (7), PHASE4 (3)
Common investigational therapies:
- Temozolomide
- Bevacizumab
- Chemotherapy
- Placebo
- Vorasidenib
- Gemcitabine
- Cyclophosphamide
- Pembrolizumab
- Irinotecan
- Thalidomide
Disease data sourced from MONDO Disease Ontology (MONDO:0021042), Orphanet — glioma, NCT00001150, NCT00001336, NCT00001341, NCT00001444, NCT00001500, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, NCT00001148, NCT00001171, NCT00001502, NCT00001573, NCT00009035, Open Targets Platform (CC BY 4.0).
Clinical development timeline
- Phase 2TBD
Phase 2 glioma study (NCT03295396)
Phase 2 clinical trial in glioma initiated; specific enrollment and design details not yet disclosed.
- Terminated2025-07-28
Program termination
Dordaviprone glioma program (ONC013) terminated by Jazz Pharmaceuticals Ireland Limited.
Competitive landscape
The glioma therapeutic landscape includes multiple approved agents, though most are indicated for other malignancies with off-label or limited glioma use. Pfizer Australia markets INLYTA (axitinib) and maintains a broad oncology portfolio. Janssen-Cilag's IMBRUVICA (ibrutinib) and Novartis's AFINITOR (everolimus) represent kinase inhibitors with potential CNS penetration but limited primary glioma indications. Jazz Pharmaceuticals itself markets VYXEOS LIPOSOMAL, a liposomal chemotherapy formulation approved for hematologic malignancies. Regeneron's LYNOZYFIC and Amgen's KYPROLIS represent additional oncology agents in the competitive space. The termination of dordaviprone suggests that despite oral bioavailability and potential CNS penetration, the compound did not demonstrate sufficient clinical benefit to warrant continued development in glioma, a highly competitive and difficult-to-treat indication where standard-of-care remains limited.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| PFIZER AUSTRALIA PTY LTD | Pfizer Australia Pty Ltd | — | approved |
| IMBRUVICA | Janssen-Cilag Pty Ltd | — | approved |
| AFINITOR | Novartis Pharmaceuticals | — | approved |
| LYSODREN | S.A. | — | approved |
| INLYTA | Pfizer Australia Pty Ltd | — | approved |
| LYNOZYFIC | Regeneron UK Limited | — | approved |
| VYXEOS LIPOSOMAL (PREVIOUSLY VYXEOS) | Jazz Pharmaceuticals Ireland Limited | — | approved |
| KYPROLIS | Amgen | — | approved |
| UNITUXIN | United Therapeutics Europe Ltd | — | approved |
| PACLITAXEL ACCORD | Accord Healthcare Pty. | — | approved |
| OFEV | Boehringer Ingelheim Pty Ltd | — | approved |
| ARX-IMATINIB | Alphapharm Pty Ltd | — | approved |
| EVEROLIMUS | — | FK506-binding protein 1A inhibitor | Approved |
| CARMUSTINE | — | Glutathione reductase inhibitor | Approved |
| BEVACIZUMAB | — | Vascular endothelial growth factor A inhibitor | Approved |
| VINCRISTINE SULFATE | — | Tubulin inhibitor | Phase 3 |
| VINCRISTINE | — | Tubulin inhibitor | Phase 3 |
| TRANEXAMIC ACID | — | Plasminogen inhibitor | Phase 3 |
| TRABEDERSEN | — | Transforming growth factor beta-2 mRNA antisense inhibitor | Phase 3 |
| TOVORAFENIB | — | RAF serine/threonine protein kinase inhibitor | Phase 3 |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
Regulatory intelligence
United States: Dordaviprone hydrochloride (MODEYSO) achieved FDA approval under NDA219876, sponsored by Chimerix, for an indication not yet disclosed in the available facts. The glioma program (ONC013) by Jazz Pharmaceuticals has been terminated and did not reach FDA filing stage.
European Union: No regulatory approval or filing status for dordaviprone in glioma has been disclosed. A related investigational immunotherapy, autologous glioma tumor cell lysates (SITOIGANAP), had its EMA application withdrawn by Epitopoietic Research Corporation-Belgium (EMEA/H/C/003693).
Japan (PMDA): Regulatory status not yet disclosed.
China (NMPA): Regulatory status not yet disclosed.
Clinical evidence summary
Key questions answered
What is dordaviprone (ONC201) used for?
Dordaviprone was being investigated for glioma treatment in a Phase 2 clinical trial by Jazz Pharmaceuticals Ireland Limited. The program has been terminated as of July 28, 2025. The approved formulation MODEYSO is indicated for a different therapeutic use by Chimerix.
Is dordaviprone approved for glioma?
No. The glioma development program was terminated in July 2025 and did not reach regulatory approval. Dordaviprone hydrochloride (MODEYSO) is FDA-approved under NDA219876 by Chimerix for a non-glioma indication.
What is the mechanism of action of dordaviprone?
The mechanism of action has not been disclosed in available regulatory or clinical documentation.
What is the molecular target of dordaviprone?
The molecular target has not been disclosed.
Who manufactures dordaviprone?
Jazz Pharmaceuticals Ireland Limited developed dordaviprone for glioma. Chimerix sponsored the FDA approval of MODEYSO (dordaviprone hydrochloride) under NDA219876.
What clinical trial supports dordaviprone in glioma?
NCT03295396 is the Phase 2 glioma trial. Specific design, enrollment, and results have not been disclosed; the trial was terminated with the program in July 2025.
What is the route of administration for dordaviprone?
Dordaviprone is administered orally.
What is the current development status of dordaviprone for glioma?
The program was terminated on July 28, 2025. No further development in glioma is planned.
What is MODEYSO?
MODEYSO is the brand name for dordaviprone hydrochloride, which is FDA-approved under NDA219876 by Chimerix for an indication not specified in available facts.
Is dordaviprone approved in Europe for glioma?
No regulatory approval or filing for dordaviprone in glioma in the European Union has been disclosed.
What competitors exist in the glioma market?
Approved agents used off-label or with limited glioma indication include AFINITOR (Novartis), IMBRUVICA (Janssen-Cilag), INLYTA (Pfizer), and others. However, no dominant standard therapy beyond chemoradiation has emerged for most gliomas.
Why was the dordaviprone glioma program terminated?
The specific reason for termination has not been disclosed. Possible factors include insufficient efficacy signals, safety concerns, or strategic portfolio prioritization by Jazz Pharmaceuticals.
Is dordaviprone still being developed for other indications?
The glioma program has been terminated. MODEYSO (dordaviprone hydrochloride) remains FDA-approved by Chimerix for its indicated use; further development status for other indications is not yet disclosed.
What is the unmet need in glioma treatment?
Glioma, particularly high-grade glioblastoma, has limited effective treatment options beyond standard chemoradiation, with poor prognosis and high mortality, representing a significant unmet medical need.
What is the drug class of dordaviprone?
Dordaviprone is classified as a small-molecule oral therapeutic. Its specific pharmacological class has not been disclosed.
When was the dordaviprone glioma program first disclosed?
The first disclosure date has not been provided in available facts.
Does Jazz Pharmaceuticals have other glioma programs?
Information regarding other Jazz Pharmaceuticals glioma programs is not provided in the available facts.
Entity relationship graph
Dordaviprone (ONC201) → Drug → Target → Indication → Company → Trials → Competitors
Companies
Indication
Trials
Analyst insights
Strategic Implications: The termination of the dordaviprone glioma program reflects Jazz Pharmaceuticals' portfolio prioritization in oncology. Despite the compound's prior FDA approval as MODEYSO by Chimerix, the glioma indication did not meet Jazz's development criteria or efficacy expectations. This suggests either insufficient Phase 2 signals or a strategic decision to focus resources on higher-probability programs.
Competitive Implications: The glioma market remains underserved with limited effective therapies beyond standard chemoradiation. The failure of dordaviprone underscores the difficulty in translating oral small-molecule efficacy from other indications into CNS malignancies. Competitors with approved agents (Pfizer, Novartis, Janssen-Cilag) maintain market presence, but no dominant therapy has emerged, indicating continued opportunity for novel mechanisms.
Future Catalysts: None anticipated for the dordaviprone glioma program given termination. Future development may focus on MODEYSO's approved indication or other Jazz Pharmaceuticals oncology assets.
- Program termination removes dordaviprone from glioma competitive landscape
- MODEYSO approval in alternate indication remains active; potential for label expansion or new indication development by Chimerix or Jazz
- Glioma market remains open for novel therapies with improved CNS penetration and efficacy
Quick answers
Concise, citable answers optimized for AI answer engines.
- What is dordaviprone?
- Small-molecule oral compound investigated for glioma; program terminated July 2025.
- Is it approved for glioma?
- No; glioma program terminated. Approved as MODEYSO by Chimerix for another indication.
- What is the indication?
- Glioma (terminated program); MODEYSO approved for undisclosed indication.
- What is the mechanism of action?
- Not yet disclosed.
- What is the molecular target?
- Not yet disclosed.
- What is the route of administration?
- Oral.
- What is the current phase?
- Terminated; was Phase 2.
- Who is the sponsor?
- Jazz Pharmaceuticals Ireland Limited (glioma program).
- What is the modality?
- Small-molecule.
- What is the internal code?
- ONC013.
- What clinical trial supports it?
- NCT03295396 (Phase 2 glioma); results not reported.
- When was the program terminated?
- July 28, 2025.
- Is there a partner?
- No partner disclosed for glioma program.
- What is MODEYSO?
- FDA-approved brand name for dordaviprone hydrochloride by Chimerix.
- What is the FDA approval status?
- MODEYSO approved under NDA219876 by Chimerix; glioma program not approved.
- What is the EMA status?
- No approval or filing disclosed for dordaviprone in glioma.
- What are key competitors?
- AFINITOR, IMBRUVICA, INLYTA, and others; no dominant glioma therapy.
- What is the therapeutic class?
- Not yet disclosed for glioma indication.
- What is the peak sales projection?
- Not yet disclosed.
- What is the consensus position?
- Not yet disclosed.
- Is there a license agreement?
- License type not disclosed for glioma program.
- What is the lead investigator?
- Not yet disclosed.
- What is the expected next milestone?
- None; program terminated.
- What is the patent status?
- Not yet disclosed.
- What is the loss of exclusivity date?
- Not yet disclosed.
- Why was the program terminated?
- Reason not disclosed; possible insufficient efficacy or safety concerns.
- What is the unmet need in glioma?
- Limited effective treatments beyond chemoradiation; high mortality.
Evidence & sources
- ClinicalTrials.gov NCT03295396 (clinicaltrials)
- dordaviprone hydrochloride US status (fda)
- autologous glioma tumor cell lysates (inactivated) EU status (ema)
- Source: phase (source_attribution)
- MONDO Disease Ontology (MONDO:0021042) (mondo)
- Orphanet — glioma (orphanet)
- NCT00001150 (clinicaltrials_gov)
- NCT00001336 (clinicaltrials_gov)
- NCT00001341 (clinicaltrials_gov)
- NCT00001444 (clinicaltrials_gov)
- NCT00001500 (clinicaltrials_gov)
- AACT (ClinicalTrials.gov aggregate) (aact)
- ClinicalTrials.gov (clinicaltrials_gov)
- NCT00001148 (clinicaltrials_gov)
- NCT00001171 (clinicaltrials_gov)
- NCT00001502 (clinicaltrials_gov)
- NCT00001573 (clinicaltrials_gov)
- NCT00009035 (clinicaltrials_gov)
- Open Targets Platform (opentargets)
Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.