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  5. Drug Trials Snapshots: VANFLYTA
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Drug Trials Snapshots: VANFLYTA

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.

Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the VANFLYTA Prescribing Information for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).

Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).

VANFLYTA (quizartinib)
van flit’ uh
Daiichi Sankyo, Inc.
Original Approval date
: July 20, 2023


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

VANFLYTA is a kinase inhibitor (a kinase is a type of protein that regulates the function of cells in your body) that is used in combination with certain chemotherapy medicines and alone as maintenance therapy to treat adults with newly diagnosed acute myeloid leukemia (AML) with a specific mutation called FLT3 internal tandem duplication (FLT3-ITD) mutation. VANFLYTA is not indicated for use alone as maintenance therapy following a stem cell transplantation (bone marrow transplant).

How is this drug used?

VANFLYTA is a tablet. One or two tablets are taken once every day by mouth. VANFLYTA can be taken with or without food.

Who participated in the clinical trials?

The FDA approved VANFLYTA based on evidence from one clinical trial of 539 patients with newly diagnosed AML. The selected patients had to have a certain type of mutation (FLT3-ITD mutation) which was confirmed using a validated assay. The trial was conducted at 193 study sites across 26 countries in North America, Europe, Asia, and other regions.

How were the trials designed?

VANFLYTA was evaluated in a clinical trial of 539 patients with newly diagnosed AML. The selected patients had to have a certain type of mutation (FLT3-ITD mutation) which was confirmed using a validated assay and had to have received no prior treatment for AML. Patients were randomly assigned to receive either VANFLYTA or placebo once a day following a specific dosing plan in combination with chemotherapy during induction and consolidation and then alone during maintenance. Neither the doctor nor the patient knew whether the patient was receiving VANFLYTA or placebo until the trial was over.

The benefit of VANFLYTA was evaluated by measuring how long and how many patients survived. The Sponsor did not know the results until the trial was completed.

DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of VANFLYTA.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 245 (45%) male patients and 294 (55%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 2 summarizes the percentage of patients by race enrolled in the clinical trial used to evaluate the efficacy of VANFLYTA.

Figure 2. Baseline Demographics by Race

Pie chart summarizing how many White, Black or African American, Asian, and other patients were in the clinical trial. In total, 322 (60%) White patients, 7 (1%) Black or African American patients, 158 (29%) Asian patients, and 52 (10%) other patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3 summarizes the percentage of patients by age group enrolled in the clinical trial used to evaluate the efficacy of VANFLYTA.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many patients by age were in the clinical trial. In total, 323 (60%) patients younger than 60 years of age, 81 (15%) patients between 60 and 65 years of age, and 135 (25%) patients 65 years of age and older participated in the clinical trial.

Source: Adapted from FDA Review

Figure 4 summarizes the percentage of patients by ethnicity enrolled in the clinical trial used to evaluate the efficacy of VANFLYTA.

Figure 4. Baseline Demographics by Ethnicity

Pie chart summarizing how many Hispanic, not Hispanic, and unknown patients were in the clinical trial. In total, 22 (4%) Hispanic or Latino patients, 470 (87%) not Hispanic or Latino patients, and 47 (9%) unknown patients participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug?

In the clinical trial, patients who received VANFLYTA in combination with chemotherapy lived longer than patients who received chemotherapy alone. The median duration of a complete remission in the 55% of patients that achieved a complete remission was 26.2 months longer when VANFLYTA was added to chemotherapy.

Were there any differences in how well the drug worked in clinical trials among sex, race and age?

  • Sex: The observed effect of VANFLYTA was similar for females and males.
  • Race: The observed effect of VANFLYTA was similar in White and Asian patients. The number of patients of races other than White and Asian was small; therefore, differences in how VANFLYTA worked among other races could not be determined.
  • Age: The observed effect of VANFLYTA was similar across all age groups.

What are the possible side effects?

VANFYTA may cause serious side effects including changes in the electrical activity of the heart called QT prolongation, irregular heart rhythm called torsade de pointes, and cause the heart to stop beating (cardiac arrest), which is life-threatening and can lead to death.

VANFLYTA may cause harm to an unborn baby (embryo-fetal toxicity).

The most common side effects of VANFLYTA are low white blood cell counts with or without fever, reduced levels of electrolytes (potassium, magnesium or calcium) in blood, increased levels of liver or muscles enzymes, diarrhea, ulcers or redness inside the mouth (mucositis), nausea, abdominal pain, sepsis (serious infection throughout the body and organs), headache, vomiting, and upper respiratory tract infection.

Were there any differences in side effects of the clinical trials among sex, race, and age?

  • Sex: The occurrence of side effects was similar in males and females.
  • Race: The occurrence of side effects was similar in White and Asian patients. The number of patients of races other than White and Asian was small; therefore, differences in the occurrence of side effects among Black or African American patients or patients of other races could not be determined.
  • Age: The occurrence of side effects was similar in patients younger and older than 65 years of age.

GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.

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