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Drug Trial Snapshot: AYVAKIT

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age 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 health provider about the risks and benefits of a drug. Refer to the  AYVAKIT Package Insert for complete information.

AYVAKIT (avapritinib)
(aye' vah kit)
Blueprint Medicines Corporation
Approval date: January 9, 2020


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

AYVAKIT is used to treat adult patients with gastrointestinal stromal tumor (GIST) whose disease:

  • is caused by certain abnormal platelet-derived growth factor receptor alpha (PDGFRA) genes and,
  • cannot be surgically removed or,
  • has spread throughout the body (metastatic GIST).

GIST is type of stomach, bowel, or esophagus tumor.

How is this drug used?

AYVAKIT is a tablet taken once daily on an empty stomach.

What are the benefits of this drug?

Thirty-six out of 43 patients (84%) experienced complete or partial shrinkage of the tumor which lasted six months or longer in 22 patients (61%).

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

  • Sex: AYVAKIT worked similarly between men and women.
  • Race: The majority of patients in the clinical trial were White. Differences among races could not be determined because of the small number of patients of other races.
  • Age: AYVAKIT worked similarly in patients younger and older than 65 years of age.

What are the possible side effects?

AYVAKIT can cause serious side effects including bleeding inside the skull, effects on the central nervous system (e.g. forgetfulness, confusion, trouble thinking, change in mood or behavior etc.) and harm to a newborn baby.

The most common side effects of AYVAKIT are swelling, nausea, tiredness, trouble thinking, vomiting, decreased appetite, diarrhea, hair color change, increased tear secretion, abdominal pain, constipation, rash and dizziness.

Were there any differences in side effects among sex, race and age?

  • Sex: The occurrence of side effects was similar in men and women.
  • Race: The majority of patients in the clinical trial were White. Differences in side effects among races could not be determined because of the small number of patients of other races.
  • Age: The occurrence of side effects was similar in patients younger and older than 65 years of age.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved AYVAKIT based on evidence from one clinical trial (NCT02508532) of 204 patients with GIST. The trial was conducted at 17 sites the United States, Europe and Asia.

Figure 1 summarizes how many men and women were in the clinical trial.

Figure 1. Baseline Demographics by Sex (safety population)

Pie chart summarizing how many men and women were in the clinical trial. In total, 80 women (39%) and 124 men (61%) participated in the clinical trial.

FDA Review

Figure 2 summarizes the percentage of patients by race in the clinical trial.

Figure 2. Baseline Demographics by Race (safety population)

Pie chart summarizing the percentage of patients by race enrolled in the clinical trial. In total, 146 White (72%), 21 Asian (10%) and Black or African American 8 (4%) and 29 Other (14%)).

*Includes American Indian or Alaska Native, Other and Unknown

FDA Review

Figure 3 summarizes how many patients of certain age were in the clinical trial.

Figure 3. Baseline Demographics by Age (safety population)

Pie charts summarizing how many individuals of certain age groups were enrolled in the clinical trial. In total,  122 (60%) were less than 65 years, and 82 (40%) of patients were 65 years and older.

FDA Review

Figure 4. Baseline Demographics by Ethnicity (safety population)

Pie charts summarizing how many individuals of certain ethnicity were enrolled in the clinical trial. In total,  6 patients were Hispanic or Latino (3%), and 172 patients were not Hispanic or Latino (84%), 26 patients were Not Reported (13%).

*includes Not Reported and Unknown

FDA Review

How were the trials designed?

There was one trial that evaluated the benefits and side effects of AYVAKIT. Patients had unresectable or metastatic GIST with mutations in PDGFRA exon 18. The most common mutation was the PDGFRA D842V mutation.

All patients received AYVAKIT once daily until disease progression or unacceptable toxicity.

The benefit of AYVAKIT was evaluated by measuring the percentage of patients who experienced partial or complete tumor shrinkage and the duration of shrinkage.

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 used in clinical trials 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.

PRESCRIBING INFORMATION

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