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Azacitidine and eltrombopag
Azacitidine is a hypomethylating agent that works by inhibiting DNA methyltransferase enzymes, leading to increased expression of tumor suppressor genes. Eltrombopag is a thrombopoietin receptor agonist that stimulates platelet production.
Azacitidine is a hypomethylating agent that works by inhibiting DNA methyltransferase enzymes, leading to increased expression of tumor suppressor genes. Eltrombopag is a thrombopoietin receptor agonist that stimulates platelet production. Used for Treatment of myelodysplastic syndromes (MDS), Treatment of low platelet count (thrombocytopenia) in patients with chronic liver disease.
At a glance
| Generic name | Azacitidine and eltrombopag |
|---|---|
| Also known as | Vidaza (azacitidine), Revolade (eltrombopag) |
| Sponsor | Peter MacCallum Cancer Centre, Australia |
| Drug class | Hypomethylating agent, Thrombopoietin receptor agonist |
| Target | DNA methyltransferase, Thrombopoietin receptor |
| Modality | Small molecule |
| Therapeutic area | Oncology, Hematology |
| Phase | Phase 2 |
Mechanism of action
Azacitidine's mechanism of action involves the inhibition of DNA methyltransferase enzymes, which are responsible for adding methyl groups to DNA, leading to gene silencing. By inhibiting these enzymes, azacitidine increases the expression of tumor suppressor genes, which helps to slow down cancer cell growth. Eltrombopag, on the other hand, works by binding to the thrombopoietin receptor, stimulating platelet production and increasing platelet counts in patients with thrombocytopenia.
Approved indications
- Treatment of myelodysplastic syndromes (MDS), Treatment of low platelet count (thrombocytopenia) in patients with chronic liver disease
Common side effects
- Nausea
- Fatigue
- Diarrhea
- Thrombocytopenia
Key clinical trials
- Decitabine for Poor Graft Function Post Allo-HSCT (PHASE3)
- Eltrombopag With Decitabine in Advanced Myelodysplastic Syndrome (MDS) (PHASE2)
- A Study of Eltrombopag or Placebo in Combination With Azacitidine in Subjects With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-risk Myelodysplastic Syndromes (MDS) (PHASE3)
- A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndromes (MDS) / Acute Myeloid Leukaemia (AML), With Eltrombopag Support for Thrombocytopenia (PHASE2)
- Safety Study of Eltrombopag Combined With Azacitidine to Treat Myelodysplastic Syndrome (MDS) (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:
- Azacitidine and eltrombopag CI brief — competitive landscape report
- Azacitidine and eltrombopag updates RSS · CI watch RSS
- Peter MacCallum Cancer Centre, Australia portfolio CI