Last reviewed 2026-04-23 · How we verify

Subcutaneous azacitidine

Azacitidine is a hypomethylating agent that works by inhibiting DNA methyltransferase enzymes, leading to increased expression of tumor suppressor genes.

Azacitidine is a hypomethylating agent that works by inhibiting DNA methyltransferase enzymes, leading to increased expression of tumor suppressor genes. Used for Treatment of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

At a glance

Mechanism of action

This results in the reactivation of silenced tumor suppressor genes, ultimately leading to the induction of apoptosis in cancer cells. Azacitidine also has immunomodulatory effects, enhancing the body's immune response against cancer cells.

Approved indications

• Treatment of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS)

Common side effects

• Fatigue
• Nausea
• Diarrhea
• Cytopenias
• Infections

Key clinical trials

• Azacitidine and Abatacept in Relapsed or Refractory T-Cell Lymphoma (PHASE1)
• Genotype-guided Targeted Agents Plus EZH2i for Primary Refractory PTCL (PHASE1, PHASE2)
• Maintenance Therapy of Hypomethylating Agent (HMA) in Favorable Risk Acute Myeloid Leukemia (AML) Patients (PHASE2)
• A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies (PHASE2)
• Azacytidine, Venetoclax Plus Minus Quizartinib for First Line Older/Unfit AML Patients (VENP-A-QUI) (PHASE3)
• Pacritinib With Aza for Upfront Myelodysplastic Syndrome (PHASE1, PHASE2)
• Ascorbate in Myelodysplastic Syndrome (PHASE2)
• A Safety Study of SEA-CD70 in Patients With Myeloid Malignancies (PHASE1)

Primary sources

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