A new DRUG TRIALS SNAPSHOT is now available.
PIQRAY is a drug for treatment of adults with a specific form of advanced breast cancer that progressed or recurred after previous hormone therapy. It is to be used in patients with:
- hormone receptor (HR) positive cancer,
- human epidermal growth factor receptor 2 (HER2)-negative cancer,
- an abnormal inherited PIK3CA gene, and
- whose cancer has spread to other parts of the body (locally advanced or metastatic)
PIQRAY is taken as two tablets once daily with food in combination with fulvestrant (another drug used to treat breast cancer).
See more Drug Trials Snapshots or contact us with questions at Snapshots@fda.hhs.gov.
Drug Trials Snapshots: PIQRAY
PIQRAY (alpelisib)
pik’ raye
Novartis Pharmaceuticals Corporation
Approval date: May 24, 2019
pik’ raye
Novartis Pharmaceuticals Corporation
Approval date: May 24, 2019
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
PIQRAY is a drug for treatment of adults with a specific form of advanced breast cancer that progressed or recurred after previous hormone therapy. It is to be used in patients with:
- hormone receptor (HR) positive cancer,
- human epidermal growth factor receptor 2 (HER2)-negative cancer,
- an abnormal inherited PIK3CA gene, and
- whose cancer has spread to other parts of the body (locally advanced or metastatic)
How is this drug used?
PIQRAY is taken as two tablets once daily with food in combination with fulvestrant (another drug used to treat breast cancer).
What are the benefits of this drug?
In patients with an abnormal inherited PIK3CA gene, those who received PIQRAY combined with fulvestrant experienced a longer time period (11 months) that tumors did not grow after treatment when compared to patients who received fulvestrant combined with placebo (5.7 months).
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: The majority of patients were women. The number of men was limited; therefore, differences in how PIQRAY worked among sex could not be determined.
- Race: The majority of patients were White. The PIQRAY worked similarly among White and Asian patients. The number of patients in other races was limited; therefore, differences in how PIQRAY worked among other races could not be determined.
- Age: PIQRAY works similarly among patients younger and older than 65 years of age.
What are the possible side effects?
PIQRAY may cause serious side effects including:
- severe allergic reactions
- severe skin reactions
- high blood sugar levels
- lung inflammation
- diarrhea
- harm to a fetus
The most common side effects of PRIQAY were high blood sugar levels (hyperglycemia), increased blood creatinine level, diarrhea, rash, increased white blood cell count, increased liver enzymes, nausea, and tiredness.
Were there any differences in side effects among sex, race and age?
- Sex: The majority of patients were women. The number of men was limited; therefore, differences in the occurrence of side effects among sex could not be determined.
- Race: The majority of patients were White. The occurrence of side effects among White and Asian patients was similar. The number of patients in other races was limited; therefore, differences in the occurrence of side effects among other races could not be determined.
- Age: There was a higher incidence of Grade 3-4 hyperglycemia (high blood sugar level) in patients older than 65 years of age when compared to patients younger than 65 years of age.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved PIQRAY based on evidence from one clinical trial (NCT02437318) of 571 patients with HR-positive, HER2-negative, advanced or metastatic breast cancer whose disease had progressed or recurred on or after hormone treatment. The trial was conducted in Asia, Australia, Canada, Europe, Latin American, and the United States.
Figure 1 summarizes how many men and women were in the clinical trial.
Figure 1. Baseline Demographics by Sex
Figure 2 summarizes the percentage of patients by race in the clinical trial.
Figure 2. Baseline Demographics by Race
*Other includes American Indian or Alaska Native and Unknown
FDA Review
Table 1. Demographics of Trial by Race
| Race | Number of Patients | Percentage of Patients |
|---|---|---|
| White | 377 | 66% |
| Black or African American | 8 | 1% |
| Asian | 125 | 22% |
| American Indian or Native Alaskan | 5 | 1% |
| Other | 26 | 5% |
| Unknown | 31 | 5% |
FDA Review
Figure 3 summarizes the percentage of patients by age in the clinical trial.
Figure 3. Baseline Demographics by Age
FDA Review
How were the trials designed?
The benefit and side effects of PIQRAY were evaluated in men and postmenopausal women with advanced HR-positive, HER2-negative breast cancer that had progressed or recurred after treatment with hormone therapy. Some patients had an abnormal PIK3CA gene.
Patients received either PIQRAY or placebo as a tablet by mouth daily in combination with fulvestrant. Fulvestrant was given as an intramuscular injection on Cycle Day 1 and Day 15 of the first 28-day cycle, and then on Cycle Day 1 of every 28-day cycle. The treatment continued until the disease progressed or the side effects became too toxic. Neither the patients nor the health care providers knew which treatment was being given until the trial was completed.
The benefit was assessed by measuring the length of time tumors did not grow after treatment (progression-free survival-PFS).
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.
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.
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