Alpelisib side effects: what 8,666 FDA adverse event reports show

Alpelisib · Piqray · 941 reaction terms · 29,096 drug–reaction co-reports · data 2026-09-14

Drug class

Kinase inhibitor

FDA label, Indications wording

Brand names

Piqray

openFDA label

Who reports

8,666 reports · 56% serious · 1,224 with death outcome · 94% female

openFDA FAERS counts, all years, deduplication not applied

Used for

PIQRAY is indicated in combination with fulvestrant for the treatment of adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer as detected by an FDA-approved test following progression on or after an endocrine-based regimen.

FDA label, Indications and Usage

What it is

PIQRAY (alpelisib) is a kinase inhibitor.

FDA label, Description section

Boxed warning

None in the current label

How to read this page. FAERS is a spontaneous-reporting system: counts are not incidence rates and a signal is not proof of causation. Each reaction below is run through an automated screen — is the term in the FDA label (known), in the indications (the disease being treated), an administrative/coding term, or none of these (unlabeled)? That screen is mechanical text matching, not a causality assessment. The 30 hand-written Bradford Hill assessments are on the causality assessment page. Not medical advice.

Causality screen of the 40 strongest signals

Labeled adverse reaction 6Labeled (related wording) 5Likely indication confounding 2Reporting / coding artifact 1Lab value / disease marker 6Not in label — unassessed 20

What the badges mean

Labeled adverse reaction The exact term appears in the label's warnings or adverse-reactions sections: a known, expected effect. The FAERS signal confirms the label rather than discovering anything.
Labeled (related wording) The label describes this effect under different wording (e.g. 'neutropenia' for 'neutrophil count decreased'). Treated as known.
Likely indication confounding The term appears in the label's indications: reports probably describe the disease being treated (or its progression), not a drug effect. Classic confounding by indication.
Reporting / coding artifact An administrative, monitoring or exposure term (product issue, off-label use, 'test normal', overdose...). Not a biological reaction; a high PRR here reflects how reports are coded.
Lab value / disease marker A laboratory result, investigation or disease-activity marker (antibody positive, CRP abnormal, swollen joint count) not described in the label. These usually mirror the monitored disease or testing protocol rather than a drug effect; not screened further.
Not in label — unassessed Not found in the label text, not an artifact pattern. This is a genuine candidate for a formal causality assessment (Bradford Hill / WHO-UMC); it has NOT had one here.

Strongest disproportionality signals

Flagged terms (PRR ≥ 2, χ² ≥ 4, n ≥ 10), ranked by PRR; infinite PRRs (no comparator reports) excluded. The EBGM column is a second, shrinkage-based estimate (MGPS) with its 5th percentile EB05 and the BCPNN IC025; a pair flagged by PRR alone is marked PRR-only and should be read as fragile. The strata column recomputes the PRR on serious reports only and flags pairs whose reports are unusually skewed by sex or come mostly from consumers rather than clinicians. 901 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.

Show: hide coding/lab/outcome terms hide indication terms unlabeled only hide PRR-only

Reaction	Reports	PRR 95% CI · ROR · χ²/p	EBGM EB05 · IC025	Label screen	Serious-only & strata	Trial cross-check
Tumour marker decreased	23	351.9 227.3–544.9ROR 352.8 · χ² 6,711 / p <1e-300	244.6 EB05 171.7 · IC025 +4.7 3/3 methods agree	Lab value / disease marker	serious n=11 · 100% consumer	no trial data cached
Pik3ca-activated mutation	37	309.9 220.1–436.2ROR 311.2 · χ² 9,811 / p <1e-300	240.4 EB05 182.1 · IC025 +5.3 3/3 methods agree	Not in label — unassessed	serious n=33	no trial data cached
Hormone receptor positive her2 negative breast cancer	21	173.4 111.4–270.0ROR 173.9 · χ² 3,198 / p <1e-300	141.2 EB05 97.5 · IC025 +4.4 3/3 methods agree	Likely indication confounding term in indications; also in warnings	serious n=21 · 90% clinician	no trial data cached
Hyperglycaemia (high blood sugar)	1,509	104.9 100.1–109.9ROR 126.8 · χ² 148,896 / p <1e-300	100.4 EB05 96.2 · IC025 +6.5 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 67.2	no trial data cached
Tumour marker abnormal	11	82.0 45.0–149.6ROR 82.1 · χ² 775 / p 8e-171	70.1 EB05 41.8 · IC025 +3.1 3/3 methods agree	Lab value / disease marker	serious n=10	no trial data cached
Carbohydrate antigen 15-3 increased	21	68.6 44.5–105.8ROR 68.7 · χ² 1,294 / p 1e-283	63.0 EB05 43.5 · IC025 +4.0 3/3 methods agree	Lab value / disease marker	serious n=21 · 90% clinician	no trial data cached
Hyperglycaemic hyperosmolar nonketotic syndrome	28	62.8 43.2–91.4ROR 63.0 · χ² 1,600 / p <1e-300	58.9 EB05 42.8 · IC025 +4.3 3/3 methods agree	Labeled adverse reaction exact term	serious n=28	no trial data cached
Breast cancer metastatic	248	57.2 50.6–64.8ROR 58.9 · χ² 13,336 / p <1e-300	55.7 EB05 50.1 · IC025 +5.4 3/3 methods agree	Likely indication confounding term in indications; also in warnings	serious PRR 58.2	no trial data cached
Metastases to pleura	22	52.2 34.2–79.6ROR 52.3 · χ² 1,032 / p 1e-226	49.1 EB05 34.2 · IC025 +3.9 3/3 methods agree	Not in label — unassessed	serious n=22 · 100% clinician	no trial data cached
Tumour marker increased	81	41.4 33.3–51.5ROR 41.8 · χ² 3,102 / p <1e-300	40.4 EB05 33.5 · IC025 +4.7 3/3 methods agree	Lab value / disease marker	serious n=53	no trial data cached
Carcinoembryonic antigen increased	31	39.5 27.7–56.2ROR 39.6 · χ² 1,106 / p 9e-243	38.0 EB05 28.0 · IC025 +4.0 3/3 methods agree	Lab value / disease marker	serious n=31	no trial data cached
Metastases to bone	181	27.1 23.4–31.3ROR 27.6 · χ² 4,470 / p <1e-300	26.7 EB05 23.6 · IC025 +4.4 3/3 methods agree	Not in label — unassessed	serious PRR 27.5	no trial data cached
Metastases to liver	175	24.4 21.0–28.3ROR 24.9 · χ² 3,865 / p <1e-300	24.1 EB05 21.2 · IC025 +4.3 3/3 methods agree	Not in label — unassessed	serious PRR 24.6	no trial data cached
Polydipsia	35	21.6 15.5–30.1ROR 21.7 · χ² 660 / p 6e-146	21.2 EB05 15.9 · IC025 +3.5 3/3 methods agree	Not in label — unassessed	serious n=31	no trial data cached
Intra-abdominal fluid collection	15	20.7 12.4–34.3ROR 20.7 · χ² 259 / p 1e-58	19.9 EB05 12.5 · IC025 +2.8 3/3 methods agree	Not in label — unassessed	serious n=14	no trial data cached
Oral mucosal eruption	11	17.5 9.7–31.7ROR 17.5 · χ² 154 / p 1e-35	16.2 EB05 8.7 · IC025 +2.3 3/3 methods agree	Not in label — unassessed	serious n=5	no trial data cached
Metastases to spine	23	16.7 11.1–25.2ROR 16.8 · χ² 322 / p 3e-72	16.2 EB05 11.1 · IC025 +2.9 3/3 methods agree	Not in label — unassessed	serious n=23	no trial data cached
Gene mutation	13	15.9 9.2–27.5ROR 16.0 · χ² 166 / p 3e-38	14.8 EB05 8.4 · IC025 +2.4 3/3 methods agree	Labeled (related wording) related wording	serious n=11	no trial data cached
Bone lesion	23	15.5 10.3–23.4ROR 15.6 · χ² 296 / p 1e-66	15.0 EB05 10.2 · IC025 +2.9 3/3 methods agree	Not in label — unassessed	serious n=22	no trial data cached
Hepatic mass	12	15.2 8.6–26.8ROR 15.2 · χ² 144 / p 2e-33	13.9 EB05 7.5 · IC025 +2.3 3/3 methods agree	Not in label — unassessed	serious n=11	no trial data cached
Growth retardation	18	14.8 9.3–23.5ROR 14.8 · χ² 217 / p 2e-49	14.0 EB05 8.9 · IC025 +2.6 3/3 methods agree	Not in label — unassessed	serious n=18	no trial data cached
Blood glucose increased	1,130	14.5 13.8–15.4ROR 16.6 · χ² 14,273 / p <1e-300	14.4 EB05 13.8 · IC025 +3.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 19.4	no trial data cached
Taste disorder	118	14.3 12.0–17.1ROR 14.5 · χ² 1,442 / p <1e-300	14.2 EB05 12.1 · IC025 +3.4 3/3 methods agree	Not in label — unassessed	serious PRR 11.9 · 91% consumer	no trial data cached
Ketoacidosis	44	14.3 10.7–19.2ROR 14.4 · χ² 529 / p 2e-117	14.0 EB05 10.8 · IC025 +3.1 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 14.1	no trial data cached
Oral pain	131	14.2 12.0–16.8ROR 14.4 · χ² 1,585 / p <1e-300	14.1 EB05 12.1 · IC025 +3.5 3/3 methods agree	Labeled (related wording) related wording	serious PRR 10.1 · 76% consumer	no trial data cached
Diabetic ketoacidosis	139	14.1 12.0–16.6ROR 14.3 · χ² 1,672 / p <1e-300	14.0 EB05 12.1 · IC025 +3.5 3/3 methods agree	Labeled (related wording) related wording	serious PRR 14.3	no trial data cached
Hepatic lesion	25	14.1 9.5–20.8ROR 14.1 · χ² 289 / p 4e-65	13.5 EB05 9.3 · IC025 +2.8 3/3 methods agree	Not in label — unassessed	serious n=23	no trial data cached
Skin toxicity	32	14.0 9.9–19.7ROR 14.0 · χ² 370 / p 1e-82	13.5 EB05 9.8 · IC025 +2.9 3/3 methods agree	Labeled (related wording) related wording	serious n=29 · 94% clinician	no trial data cached
Blood glucose abnormal	106	13.9 11.5–16.8ROR 14.1 · χ² 1,253 / p 1e-274	13.8 EB05 11.7 · IC025 +3.4 3/3 methods agree	Lab value / disease marker	serious PRR 21.5	no trial data cached
Stomatitis (mouth sores)	343	13.8 12.4–15.3ROR 14.3 · χ² 4,032 / p <1e-300	13.7 EB05 12.5 · IC025 +3.6 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 9.6 · 72% consumer	no trial data cached
Psychiatric decompensation	10	12.5 6.7–23.2ROR 12.5 · χ² 94 / p 2e-22	10.4 EB05 5.3 · IC025 +1.9 3/3 methods agree	Not in label — unassessed	serious n=10	no trial data cached
Metastases to lung	59	12.4 9.6–16.0ROR 12.5 · χ² 606 / p 5e-134	12.2 EB05 9.7 · IC025 +3.1 3/3 methods agree	Not in label — unassessed	serious PRR 12.5	no trial data cached
Malignant neoplasm progression	511	12.2 11.2–13.3ROR 12.9 · χ² 5,239 / p <1e-300	12.1 EB05 11.3 · IC025 +3.4 3/3 methods agree	Reporting / coding artifact	serious PRR 12.5	no trial data cached
Mucosal dryness	10	12.2 6.5–22.6ROR 12.2 · χ² 91 / p 7e-22	10.1 EB05 5.1 · IC025 +1.9 3/3 methods agree	Labeled adverse reaction exact term	serious n=9	no trial data cached
Feeding disorder	98	11.9 9.8–14.6ROR 12.1 · χ² 968 / p 7e-213	11.8 EB05 9.9 · IC025 +3.2 3/3 methods agree	Not in label — unassessed	serious PRR 10.6 · 79% consumer	no trial data cached
Metastasis	34	11.5 8.2–16.1ROR 11.6 · χ² 315 / p 1e-70	11.0 EB05 8.0 · IC025 +2.7 3/3 methods agree	Not in label — unassessed	serious PRR 11.9	no trial data cached
Lip blister	12	10.7 6.1–18.8ROR 10.7 · χ² 95 / p 8e-23	8.9 EB05 5.0 · IC025 +2.0 3/3 methods agree	Not in label — unassessed	serious n=3	no trial data cached
Oral mucosal blistering	27	10.3 7.0–15.0ROR 10.3 · χ² 215 / p 5e-49	9.5 EB05 6.6 · IC025 +2.5 3/3 methods agree	Not in label — unassessed	serious n=13 · 70% consumer	no trial data cached
Metastases to lymph nodes	28	10.1 7.0–14.7ROR 10.2 · χ² 221 / p 3e-50	9.4 EB05 6.6 · IC025 +2.5 3/3 methods agree	Not in label — unassessed	serious n=28	no trial data cached
Thirst	73	9.9 7.9–12.5ROR 10.0 · χ² 576 / p 1e-127	9.6 EB05 7.9 · IC025 +2.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 11.3 · 79% consumer	no trial data cached

Trial evidence (ClinicalTrials.gov)

Trial cross-check not yet run for this drug (ClinicalTrials.gov pull covers the 800 most-reported drugs first).

Most frequently reported reactions

By raw co-report count, regardless of disproportionality. Common terms here are often the illness itself or generic outcomes.

Reaction	Reports	PRR 95% CI · ROR · χ²/p	EBGM EB05 · IC025	Label screen	Serious-only & strata	Trial cross-check
Hyperglycaemia (high blood sugar)	1,509	104.9 100.1–109.9ROR 126.8 · χ² 148,896 / p <1e-300	100.4 EB05 96.2 · IC025 +6.5 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 67.2	no trial data cached
Rash	1,391	7.8 7.4–8.2ROR 9.1 · χ² 8,364 / p <1e-300	7.7 EB05 7.4 · IC025 +2.9 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 8.2	no trial data cached
Diarrhoea (diarrhea)	1,356	5.1 4.9–5.4ROR 5.9 · χ² 4,610 / p <1e-300	5.1 EB05 4.9 · IC025 +2.3 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 4.4	no trial data cached
Blood glucose increased	1,130	14.5 13.8–15.4ROR 16.6 · χ² 14,273 / p <1e-300	14.4 EB05 13.8 · IC025 +3.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 19.4	no trial data cached
Death	896	2.5 2.4–2.7ROR 2.7 · χ² 853 / p 7e-188	2.5 EB05 2.4 · IC025 +1.2 3/3 methods agree	Outcome field, not a reaction	serious PRR 2.6	no trial data cached
Nausea	861	2.6 2.5–2.8ROR 2.8 · χ² 911 / p 2e-200	2.6 EB05 2.5 · IC025 +1.3 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 2.6	no trial data cached
Fatigue (tiredness)	852	2.7 2.5–2.8ROR 2.8 · χ² 911 / p 2e-200	2.7 EB05 2.5 · IC025 +1.3 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 2.6	no trial data cached
Decreased appetite (loss of appetite)	548	5.7 5.3–6.2ROR 6.1 · χ² 2,157 / p <1e-300	5.7 EB05 5.3 · IC025 +2.4 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 4.6 · 71% consumer	no trial data cached
Weight decreased (weight loss)	526	4.6 4.2–5.0ROR 4.8 · χ² 1,479 / p <1e-300	4.5 EB05 4.2 · IC025 +2.0 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 4.1	no trial data cached
Malignant neoplasm progression	511	12.2 11.2–13.3ROR 12.9 · χ² 5,239 / p <1e-300	12.1 EB05 11.3 · IC025 +3.4 3/3 methods agree	Reporting / coding artifact	serious PRR 12.5	no trial data cached
Vomiting	489	2.5 2.3–2.8ROR 2.6 · χ² 459 / p 4e-102	2.5 EB05 2.3 · IC025 +1.2 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 2.4	no trial data cached
Drug ineffective (drug did not work)	382	0.7 0.6–0.8ROR 0.7 · χ² 51 / p 4e-13	0.7 EB05 0.6 · IC025 -0.7 PRR-only: not supported by EBGM or IC	Reporting / coding artifact	serious PRR 1.3	no trial data cached
Asthenia (weakness)	346	2.2 2.0–2.5ROR 2.3 · χ² 237 / p 9e-54	2.2 EB05 2.0 · IC025 +1.0 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 2.1	no trial data cached
Stomatitis (mouth sores)	343	13.8 12.4–15.3ROR 14.3 · χ² 4,032 / p <1e-300	13.7 EB05 12.5 · IC025 +3.6 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 9.6 · 72% consumer	no trial data cached
Pruritus (itching)	281	1.8 1.6–2.0ROR 1.8 · χ² 101 / p 5e-24	1.8 EB05 1.6 · IC025 +0.6	Labeled adverse reaction exact term	serious PRR 2.9	no trial data cached

Unlabeled candidate signals

These flagged terms were not found in the FDA label text and do not match an artifact pattern. They are the pairs that would merit a structured assessment (temporality, dose-response, biological plausibility, consistency across databases, trial or cohort evidence). The screen has not done that; string-matching misses label wording, and a strong PRR can still be stimulated reporting, co-medication, or the disease itself.

Pik3ca-activated mutation — n=37, PRR 309.9
Metastases to pleura — n=22, PRR 52.2
Metastases to bone — n=181, PRR 27.1
Metastases to liver — n=175, PRR 24.4
Polydipsia — n=35, PRR 21.6
Metastases to spine — n=23, PRR 16.7
Bone lesion — n=23, PRR 15.5
Taste disorder — n=118, PRR 14.3
Hepatic lesion — n=25, PRR 14.1
Metastases to lung — n=59, PRR 12.4

Want to assess one of these properly? The runbook describes the Bradford Hill / trial-evidence workflow used on the assessment page, and handoff_top1000_signals.csv lists the strongest unassessed pairs across all drugs.

Common questions

What are the most reported side effects of Alpelisib?

In FDA FAERS, the most frequently co-reported reaction terms for Alpelisib are hyperglycaemia, rash, diarrhoea, blood glucose increased, nausea, fatigue. These are report counts, not rates: FAERS has no denominator, reporting is voluntary and uneven, and a term can be reported because of the illness being treated rather than the drug.

Does Alpelisib have an FDA boxed warning?

No. The FDA label retrieved for Alpelisib has no boxed warning; see the label's Warnings and Precautions for the recognised risks.

Do FAERS signals prove that Alpelisib causes these side effects?

No. A disproportionality signal (PRR, ROR) means a reaction is reported more often with this drug than with others in the database. That can reflect a real effect, but also the disease being treated, stimulated reporting after news or litigation, co-medications, or how reports are coded. Causality needs temporality, dose-response, biological plausibility, consistency across data sources and, ideally, trial or cohort evidence. This page screens each term against the FDA label and ClinicalTrials.gov results; it does not establish causation.

Sources & methods

FDA label via openFDA (set ID b20b4e18-7a4b-4500-a08f-06c6dab0ee5b, effective 20251215; matched on openfda.generic_name.exact:"ALPELISIB")
Bayesian shrinkage measures computed offline over all 1,317,300 cached pairs: BCPNN information component (IC, with Norén closed-form IC025) and MGPS empirical-Bayes geometric mean (EBGM, EB05) with a two-component gamma prior fitted by maximum marginal likelihood (α₁=0.57, β₁=0.02, α₂=1.42, β₂=0.70, w=0.17); unstratified
FAERS pair statistics from the OpenPV build of 2026-09-14 (openFDA count= aggregation, field patient.drug.openfda.generic_name.exact, 941 reaction terms, min 3 co-reports); PRR/ROR with 95% CI, chi-square with Yates correction
Serious-only and stratified counts (serious:1, patient.patientsex, primarysource.qualification) from openFDA count queries; serious-only PRR uses the drug serious report total and the term global serious total (N_serious=11,882,979); strata shown only when n ≥ 20
Seriousness and sex counts from openFDA count=serious, patient.patientsex, seriousnessdeath

Page generated 2026-10-08. Label and PubChem text are retrieved automatically and may lag the current label; always check the current prescribing information. OpenPV is a beta research tool of the Open Source Medicine Foundation.