Azafab is an antineoplastic agent that works as a hypomethylating agent. It is primarily used in the treatment of certain types of myelodysplastic syndromes (MDS), a group of bone marrow disorders. Azafab helps restore normal gene function, promoting healthy blood cell production and inhibiting the growth of abnormal cells in the bone marrow.

Indications:

1. Myelodysplastic Syndromes (MDS):
   • Refractory anemia or anemia with excess blasts.
   • Chronic myelomonocytic leukemia (CMML).
   • Intermediate-2 and high-risk MDS based on the International Prognostic Scoring System (IPSS).
2. Acute Myeloid Leukemia (AML):
   • For patients with AML who are not candidates for intensive chemotherapy.

Azafab incorporates into DNA and RNA, inhibiting DNA methyltransferase. This results in hypomethylation of DNA and reactivation of silenced tumor suppressor genes. The drug also disrupts RNA processing, which may contribute to its anti-cancer effects. These mechanisms slow or stop the growth of abnormal cells and promote normal cell development.