Acute Myeloid Leukemia (AML) Medications List

AML Medications

Acute Myeloid Leukemia (AML) is a type of cancer that affects the bone marrow and blood cells. It occurs due to abnormal myeloid cells which produce RBCs & WBCs.

This type of cancer is more common in adults over 65 years of age. The chances of survival mainly depend on early diagnosis and proper treatment.

Here is a list of effective AML medications.



Nucleoside metabolic inhibitors
  • Azacitidine
  • Decitabine
Anthracycline antibiotics
  • Daunorubicin
  • Idarubicin
Purine analogs
  • Fludarabine
  • Cladribine
Nucleoside analogsCytarabine
Alkylating agentsCyclophosphamide
Monoclonal antibodiesGemtuzumab
Protein kinase inhibitorsMidostaurin
Tyrosine kinase inhibitorsGilteritinib
IDH inhibitors (1 & 2)
  • Ivosidenib
  • Olutasidenib
  • Enasidenib
BCL 2 inhibitorsVenetoclax
Hedgehog pathway inhibitorsGlasdegib

Azacitidine for AML

Generic name: Azacitidine

Trade name: Onureg, Vidaza

Date of approval: September 1, 2020 [ref]

Company name: Reddy’s Laboratories


For the therapy of Acute Myeloid Leukemia, Refractory Anemia, and Chronic Myelomonocytic Leukemia.


A chemical analog of the cytosine nucleoside found in DNA and RNA is azacitidine (5-azacytidine). Azacitidine has the potential to cause cytotoxicity by incorporation into RNA and DNA at high dosages and antineoplastic action by inhibiting DNA methyltransferase at low concentrations.

DNA becomes hypomethylated as a result of covalent attachment to DNA methyltransferase, which also stops DNA synthesis.

As a ribonucleoside, azacitidine incorporates more readily into RNA than DNA. As a result of the incorporation into RNA, polyribosomes disassemble, transfer RNA’s methylation and acceptor function are compromised, protein synthesis is inhibited, and the cell dies.

Major contraindications:


  • Hypersensitivity to azacitidine or its ingredients

SC or IV

  • Hypersensitivity to azacitidine or mannitol
  • Advanced malignant hepatic tumors


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 Cytarabine for AML

Generic name: Cytarabine

Trade name: Cytosar, Vyxeos

Company name: Pfizer Inc.

Date of approval: August 3, 2017 [ref]


Direct DNA damage and integration into DNA are how cytarabine works. It appears to function by inhibiting DNA polymerase, while the exact mechanism of action is yet unclear. There has also been evidence of a small but considerable incorporation of cytarabine into both DNA and RNA.


Acute lymphoblastic leukemia, acute myeloid leukemia, and chronic myeloid leukemia are conditions for which Cytarabine is occasionally used.

Major contraindications:

Hypersensitivity, ongoing meningeal infection

Daunorubicin for AML

Generic name: Daunorubicin

Trade name: Cerubidine

Date of approval: December 19, 1979 [ref]

Company name: Hikma Pharmaceuticals


For the therapy of acute myeloid leukemia, and acute lymphocytic leukemia in children.


This anthracycline antibiotic, daunorubicin harms DNA by intercalating between base pairs, which causes the helix to uncoil and ultimately prevents DNA synthesis and DNA-dependent RNA synthesis.

Major contraindications:

  • Active infection
  • Patients who have taken the maximum cumulative dosage of daunorubicin/doxorubicin
  • Hypersensitivity
  • IM or SC administration (vesicant)
  • Patients with already existing myelosuppression due to other drug therapies
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Idarubicin for AML

Generic name: Idarubicin

Trade name: Idamycin

Date of approval: September 1990 [ref]

Company name: Pfizer Oncology


For the therapy of acute myeloid leukemia.


Idarubicin intercalates into DNA and disrupts the function of topoisomerase II, preventing the replication of DNA, transcription of RNA, and synthesis of proteins.

Idarubicin penetrates cell membranes more effectively than other anthracycline antibiotic compounds because of its high lipophilicity.

Major contraindications:

  • Serum bilirubin above 5 mg/dL
  • Hypersensitivity

Fludarabine for AML

Generic name: Fludarabine

Trade name: Fludara

Company name: Bayer HealthCare Pharmaceuticals

Date of approval: April 18, 1991 [ref]


Fludarabine phosphate undergoes quick dephosphorylation to become 2-fluoro-ara-A, which is subsequently intracellularly phosphorylated by deoxycytidine kinase to become the active triphosphate, 2-fluoro-ara-ATP.

This metabolite appears to prevent DNA synthesis by impeding DNA primase, ribonucleotide reductase, and DNA polymerase alpha. This drug’s mode of action is not fully understood and could have many components.


In order to treat chronic lymphocytic leukemia (CLL), fludarabine is typically utilized. Acute myeloid leukemia, hairy cell leukemia, low-grade non-Hodgkin lymphoma (NHL), and mycosis fungoides, a skin-related malignancy, may also be treated with it in clinical studies.

Major contraindications:

  • ​​Hypersensitivity
  • Concurrent use of pentostatin: chances of deadly pulmonary toxicity.
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Cyclophosphamide for AML

Generic name: Cyclophosphamide

Trade name:  Procytox, Cytoxan

Company name: Baxter Oncology

Date of approval: August 17, 1999 [ref]


Cyclophosphamide is a nitrogen mustard-type alkylating agent. Cross-linking of DNA and RNA strands and the suppression of protein synthesis are the principal causes of its cytotoxic effects.


It is used for the therapy of multiple myeloma,  acute myelogenous and monocytic leukemia [fda indications], mycosis fungoides, malignant lymphomas, retinoblastoma, neuroblastoma, and adenocarcinoma of the ovary.

Major contraindications:

  • Hypersensitivity
  • Severe myelosuppression
  • Urinary outflow obstruction

Cladribine Decitabine For AML

Generic name: Cladribine

Trade name: Litak, Mavenclad

Date of approval: Not available for AML

Company name: Merck


For the therapy of Non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia, and Acute Myeloid Leukemia.


By incorporating themselves into DNA and by inhibiting enzymes involved in DNA metabolism, such as DNA polymerase and ribonucleotide reductase, cladribine-phosphates prevent the synthesis and repair of DNA. DNA strand breakage and ultimately cell death result from this.

Major contraindications:


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Decitabine for AML

Generic name: Decitabine

Trade name: Dacogen, Inqovi 5 Tablet Pack

Date of approval: Not available for AML

Company name: Genentech Inc


As a first-line treatment for elderly AML patients


Causes hypomethylation of DNA and cellular differentiation or death by inhibiting DNA methyltransferase

Major contraindications:


Gemtuzumab for AML

Generic name: Gemtuzumab

Trade name: Mylotarg

Date of approval: September 1, 2017 [ref]

Company name: Pfizer


It is recommended for the treatment of individuals with CD33-positive acute myeloid leukemia in the first relapse who are 60 or over in age and who are not deemed suitable for other cytotoxic chemotherapy.

Indicated for the treatment of individuals with CD33-positive AML who are 2 years of age or older and have had a recurrence or have not responded to initial therapy (refractory).


Mylotarg works by targeting the CD33 antigen that hematopoietic cells express. A complex that is internalized is created when the anti-CD33 antibody component of Mylotarg binds to the CD33 antigen.

The calicheamicin derivative is internalized and then released inside the myeloid cell’s lysosomes.

Through the production of a p-benzene diradical, the calicheamicin derivative that has been produced attaches to DNA in the minor groove and causes site-specific DNA double-strand breaks. Ultimately, cell death is brought about.

Major contraindications:

Hypersensitivity to the gemtuzumab active ingredient, any of its ingredients, or any of the excipients

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Midostaurin for AML

Generic name: Midostaurin

Trade name: Rydapt

Date of approval: April 28, 2017 [ref]

Company name: Novartis


For use in adult patients with FLT3 mutation-positive high-risk acute myeloid leukemia (AML), systemic mastocytosis with associated hematological neoplasm (SM-AHN), and severe systemic mastocytosis (ASM).


It effectively blocks a number of receptor tyrosine kinases. A number of tyrosine kinases, including protein kinase C alpha (PKCalpha), VEGFR2, KIT, PDGFR, and WT and/or mutant FLT3 tyrosine kinases are inhibited by midostaurin and its two main active metabolites, CGP62221 and CGP52421.

Apoptosis is induced in target leukemia cells that express the target receptors as well as in mast cells when FLT3 receptor signaling cascades are inhibited. This is in addition to the antiproliferative effect against various cancer cell lines.

Major contraindications:

Hypersensitivity responses have included angioedema, dyspnea, flushing, and anaphylactic shock

Gilteritinib for AML


Generic name: Gilteritinib

Trade name: Xospata

Date of approval: November 28, 2018 [ref]

Company name: Astellas Pharma


Adult patients with acute myeloid leukemia who have relapsed or are resistant to therapy and have an FLT3 mutation identified by an FDA-approved test are eligible for the use of gilteritinib.


The FLT3 receptor’s internal tandem duplication (ITD) and tyrosine kinase domain (TKD) mutations may both be inhibited by gilteritinib, which has a strong selective inhibitory effect.

AXL and ALK tyrosine kinases are likewise inhibited by gilteritinib, it is also stated. Cancer cell development is influenced by the molecules FLT3 and AXL.

The phosphorylation of FLT3 and its downstream targets, such as STAT5, ERK, and AKT, can be inhibited by gilteritinib’s action.

Major contraindications:

Hypersensitivity towards this drug or any of the excipients

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Glasdegib for AML

Generic name: Glasdegib

Trade name: Daurismo

Date of approval: November 21, 2018 [ref]

Company name: Pfizer Inc.


Adult patients with newly diagnosed acute myeloid leukemia who are over 75 years old or who have co-morbidities that prevent rigorous induction chemotherapy are recommended to receive glasdegib in conjunction with cytarabine.


Disrupts the hedgehog pathway by inhibiting the smoothen (SMO) receptor, a transmembrane protein involved in hedgehog (Hh) signal transmission.

SMO suppression of hedgehog signaling alters the control of cancer stem cell survival, which has an influence on tumor biology. This might hinder the emergence of medication resistance and stop relapse in AML patients.

Major contraindications:


Venetoclax for AML

Generic name: Venetoclax

Trade name: Venclexta

Date of approval: October 16, 2020 [ref]

Company name: AbbVie Inc.


For the treatment of adult patients with small lymphocytic lymphoma (SLL) or chronic lymphocytic leukemia (CLL), venetoclax is recommended.

Additionally, it is used in conjunction with azacitidine, decitabine, or low-dose cytarabine to treat newly diagnosed acute myeloid leukemia (AML) in people 75 years of age or older, or in those who have comorbidities that prevent the administration of aggressive induction chemotherapy.


This medication is a selective inhibitor of the anti-apoptotic protein, which regulates B-cell lymphoma 2.

By directly attaching to the Bcl-2 protein, displacing proapoptotic proteins including BIM (a new member of the Bcl-2 family), inducing mitochondrial outer membrane permeabilization, and activating caspases, venetoclax aids in the restoration of the apoptotic process.

Major contraindications:

Strong CYP3A inhibitors at the start and during the ramp-up phase

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Enasidenib for AML

Generic name: Enasidenib

Trade name: Idhifa

Date of approval: August 1, 2017 [ref]

Company name: Agios Pharmaceuticals


Indicated for the treatment of adult patients with isocitrate dehydrogenase-2 (IDH2) mutation-positive relapsed or refractory acute myeloid leukemia (AML).


This IDH2 inhibitor is an oral, reversible, selective IDH2 inhibitor. It blocks the activity of mutant IDH2, which lowers levels of 2-hydroxyglutarate (2-HG) and promotes myeloid differentiation in vitro.

Enasidenib’s ability to decrease 2-HG levels and induce blast differentiation may allow it to show therapeutic effectiveness in AML with IDH2 mutations.

Major contraindications:


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Ivosidenib: One of the latest FDA-Approved AML Medications

Generic name: Ivosidenib

Trade name: Tibsovo

Date of approval: May 2, 2019 [ref]

Company name: Servier Pharmaceuticals LLC


Ivosidenib is an FDA-approved isocitrate dehydrogenase-1 (IDH1) inhibitor that is prescribed for the treatment of individuals with the following symptoms of a vulnerable IDH1 mutation:

  • For the treatment of newly diagnosed acute myeloid leukemia (AML) in persons 75 years of age or older, or who have comorbidities that prevent the use of intense induction chemotherapy, it may be used alone or in conjunction with azacitidine.
  • Adults with refractory or relapsed AML.
  • Adults who have previously had treatment for cholangiocarcinoma with localized advanced or metastatic disease.


Isocitrate dehydrogenase-1 inhibitor Tibsovo (ivosidenib) prevents cancer cells from differentiating by reducing the excessive synthesis of the oncometabolite 2-hydroxyglutarate (2-HG).

Major contraindications:


Olutasidenib: One of the latest FDA-Approved AML Medications

Generic name: Olutasidenib

Trade name: Rezlidhia

Date of approval: December 1, 2022 [ref]

Company name: Rigel Pharmaceuticals


Adult patients with relapsed or resistant acute myeloid leukemia (AML) who have an FDA-approved test-detected susceptible isocitrate dehydrogenase-1 (IDH1) mutation should be treated with olutasidenib.


Olutasidenib particularly inhibits IDH1(R132) upon delivery, preventing the oncometabolite 2-hydroxyglutarate (2HG) from being produced from alpha-ketoglutarate (a-KG).

Major contraindications:


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Written by Dr. Ahmed

I am Dr. Ahmed (MBBS; FCPS Medicine), an Internist and a practicing physician. I am in the medical field for over fifteen years working in one of the busiest hospitals and writing medical posts for over 5 years.

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