The path to approval for oral hypomethylating agents in acute myeloid leukemia and myelodysplastic syndromes.

Abstract

Two oral hypomethylating agents, oral azacitidine (CC-486) and decitabine/cedazuridine (ASTX727), have recently entered the clinical domain. CC-486 has been shown to improve overall survival as maintenance therapy for older patients with acute myeloid leukemia in complete remission, whereas the combination of decitabine with cedazuridine, a cytidine deaminase inhibitor, is indicated for the treatment of adult patients with myelodysplastic syndromes and chronic myelomonocytic leukemia with intermediate-1, or higher, International Prognostic Scoring System risk. This article briefly summarizes the clinical development of both drugs, the pivotal studies that led to their approval and some of the issues faced in extending the use of these drugs to other indications.

• Authors: David Kipp, Andrew H Wei
• Journal: Future oncology (London, England) (Future Oncol) Vol. 17 Issue 20 Pg. 2563-2571 (Jul 2021) ISSN: 1744-8301 [Electronic] England
• PMID: 33769069 (Publication Type: Journal Article)

Chemical References

• Antimetabolites, Antineoplastic
• Drug Combinations
• cc-486
• decitabine and cedazuridine drug combination
• cedazuridine
• Decitabine
• Azacitidine
• Uridine

Topics

• Antimetabolites, Antineoplastic (administration & dosage, adverse effects, pharmacokinetics)
• Azacitidine (administration & dosage, adverse effects, pharmacokinetics)
• Biological Availability
• Clinical Trials, Phase III as Topic
• DNA Methylation (drug effects)
• Decitabine (administration & dosage, adverse effects, pharmacokinetics)
• Drug Approval
• Drug Combinations
• Humans
• Leukemia, Myeloid, Acute (drug therapy)
• Myelodysplastic Syndromes (drug therapy)
• Randomized Controlled Trials as Topic
• Remission Induction (methods)
• Uridine (administration & dosage, adverse effects, analogs & derivatives, pharmacokinetics)