Targeted Therapy for Acute Myeloid Leukemia

Targeted therapy uses drugs to target specific changes in cancer cells that help them grow, divide, and spread. Targeted therapy drugs are designed to be more precise. They fight cancer cells while causing less harm to other cells in the body.

Most targeted therapies for AML are taken by mouth as a pill. Some targeted drugs may be given by IV (through a vein), sometimes in combination with chemotherapy.

These are the latest targeted therapies available to treat AML. New treatments become available all the time, so this may not be a complete list.

Please note: The side effects listed here do not represent a comprehensive list. It's important to discuss all potential side effects of a drug with your healthcare team. Be sure to tell your healthcare team about any side effects you do have.

All Available Treatments

Intravenous =
Pill =

Treatment Type

FLT3 Inhibitor

Drug Names
Rydapt® (midostaurin) (Pill)
Vanflyta® (quizartinib) (Pill)
Xospata® (gilteritinib) (Pill)
Potential Severe Side Effects
Liver problems Kidney problems
Potential Side Effects - All Drugs
Diarrhea Fatigue/weakness Headache Joint pain/stiffness Kidney problems Low blood counts Mouth/throat changes Muscle pain Nausea/vomiting Swelling
Side Effects - Vanflyta Only
Abnormal lab tests Infections
Important Things to Know
  • This treatment is only given if the patient has a FLT3 gene mutation.
  • Low blood counts are a potential side effect that may put you at risk for anemia, infection, or bleeding.
Important Things to Know - Vanflyta Only
  • These medications may cause fetal harm 
  • These medications may cause cardiovascular problems including heart attack 
  • Vanflyta® (quizartinib) is given in combination with chemotherapy

Treatment Type

IDH Inhibitor

Drug Names
Idhifa® (enasidenib) (Pill)
Rezlidhia® (olutasidenib) (Pill)
Tibsovo® (ivosidenib) (Pill)
Potential Side Effects
Diarrhea Electrolyte imbalance Fatigue/weakness Joint pain/stiffness Low blood counts Nausea/vomiting Swelling Weight/appetite changes
Important Things to Know
  • For patients who have a targetable IDH1 or IDH2 gene mutation.
  • A potentially life-threatening side effect is differentiation syndrome.
  • Low blood counts are a potential side effect that may put you at risk for anemia, infection, or bleeding.

Treatment Type

Anti-CD33 Antibody Conjugate

Drug Name
Mylotarg® (gemtuzumab ozogamicin) (IV)
Potential Severe Side Effects
Liver problems
Potential Side Effects
Fatigue/weakness Fever Low blood counts Nausea/vomiting
Important Things to Know
  • May be recommended if patient’s cancer type tests positive for the CD33 protein on the cancer cell surface.
  • Low blood counts are a potential side effect that may put you at risk for anemia, infection, or bleeding.

Treatment Type

BCL-2 Inhibitor

Drug Name
Venclexta® (venetoclax) (Pill)
Potential Severe Side Effects
Tumor Lysis Syndrome
Potential Side Effects
Constipation Cough Diarrhea Fever Low blood counts Nausea/vomiting Swelling
Important Things to Know
  • This is a treatment type for an AML cancer type that expresses the BCL-2 protein.
  • Only indicated for use in combination with low-dose chemotherapy, and also in patients 75 years of age or older or those of any adult age who are not healthy enough to receive intensive chemotherapy.
  • Low blood counts are a potential side effect that may put you at risk for anemia, infection, or bleeding.

Treatment Type

Hedgehog Pathway Inhibitor

Drug Name
Daurismo® (glasdegib) (Pill)
Potential Side Effects
Electrolyte imbalance Fatigue/weakness Fever Joint pain/stiffness Low blood counts Muscle pain Nausea/vomiting Swelling
Important Things to Know
  • Only indicated for use in patients 75 years of age or older or those of any adult age who are not healthy enough to receive intensive chemotherapy and is taken in combination with low-dose cytarabine.
  • Low blood counts are a potential side effect that may put you at risk for anemia, infection, or bleeding.