Mitapivat side effects: what 1,017 FDA adverse event reports show

Mitapivat · Aqvesme · 14 reaction terms · 485 drug–reaction co-reports · data 2026-09-14

Drug class

Pyruvate Kinase Activator

FDA label (Established Pharmacologic Class)

Brand names

Aqvesme

openFDA label

Who reports

1,017 reports · 16% serious · 6 with death outcome · 59% female

openFDA FAERS counts, all years, deduplication not applied

Used for

AQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. AQVESME is a pyruvate kinase activator indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. ( 1.1 )

FDA label, Indications and Usage

What it is

The active ingredient of AQVESME is mitapivat, a pyruvate kinase activator, present as mitapivat sulfate.

FDA label, Description section

Boxed warning

HEPATOCELLULAR INJURY AQVESME can cause serious hepatocellular injury. Measure liver laboratory tests (ALT, AST, alkaline phosphatase, and total bilirubin with fractionation) at baseline and every 4 weeks for 24 weeks and then as clinically indicated. Avoid use of AQVESME in patients with cirrhosis.

FDA 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 12 strongest signals

Labeled — boxed warning 1Labeled adverse reaction 1Reporting / coding artifact 3Lab value / disease marker 2Not in label — unassessed 5

What the badges mean

Labeled — boxed warning The term (or its clinical synonym) appears in the FDA boxed warning: causality is regulator-recognised and considered serious.
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.
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. 2 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
Haemoglobin decreased	211	42.4 37.6–47.8ROR 53.2 · χ² 8,504 / p <1e-300	42.1 EB05 37.6 · IC025 +5.0 3/3 methods agree	Lab value / disease marker	serious PRR 42.4 · 86% consumer	no trial data cached
Product dose omission in error	37	28.4 20.7–39.0ROR 29.5 · χ² 952 / p 2e-209	28.0 EB05 21.2 · IC025 +3.8 3/3 methods agree	Reporting / coding artifact	serious PRR 21.4 · 100% consumer	no trial data cached
Brain fog	13	18.4 10.7–31.6ROR 18.6 · χ² 197 / p 6e-45	17.5 EB05 10.3 · IC025 +2.5 3/3 methods agree	Not in label — unassessed	serious PRR 9.6	no trial data cached
Chromaturia	16	14.6 9.0–23.8ROR 14.8 · χ² 190 / p 2e-43	13.8 EB05 8.3 · IC025 +2.5 3/3 methods agree	Not in label — unassessed	–	no trial data cached
Initial insomnia	7	14.2 6.8–29.6ROR 14.3 · χ² 73 / p 7e-18	11.2 EB05 4.7 · IC025 +1.6 3/3 methods agree	Labeled adverse reaction exact term	–	no trial data cached
Impaired work ability	12	11.5 6.6–20.3ROR 11.7 · χ² 105 / p 5e-25	9.8 EB05 5.4 · IC025 +2.0 3/3 methods agree	Not in label — unassessed	serious PRR 4.3	no trial data cached
Jaundice (yellowing of skin/eyes)	15	11.5 6.9–19.0ROR 11.6 · χ² 133 / p 4e-31	10.2 EB05 6.0 · IC025 +2.2 3/3 methods agree	Labeled — boxed warning exact term	serious PRR 5.7	no trial data cached
Product dose omission issue	138	10.0 8.6–11.7ROR 11.4 · χ² 1,123 / p 1e-246	9.9 EB05 8.5 · IC025 +3.0 3/3 methods agree	Reporting / coding artifact	serious PRR 30.8 · 88% consumer	no trial data cached
Full blood count abnormal	5	9.8 4.1–23.5ROR 9.8 · χ² 31 / p 1e-8	6.2 EB05 2.6 · IC025 +0.9 3/3 methods agree	Lab value / disease marker	serious PRR 9.2	no trial data cached
Laboratory test abnormal	12	8.4 4.8–14.7ROR 8.5 · χ² 71 / p 2e-17	6.8 EB05 4.0 · IC025 +1.7 3/3 methods agree	Reporting / coding artifact	–	no trial data cached
Thirst	7	8.1 3.9–16.9ROR 8.1 · χ² 37 / p 7e-10	5.8 EB05 2.9 · IC025 +1.2 3/3 methods agree	Not in label — unassessed	serious PRR 15.1	no trial data cached
Sickle cell anaemia with crisis	5	6.5 2.7–15.6ROR 6.6 · χ² 18 / p 1e-5	4.4 EB05 2.0 · IC025 +0.6 3/3 methods agree	Not in label — unassessed	serious PRR 32.1	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
Haemoglobin decreased	211	42.4 37.6–47.8ROR 53.2 · χ² 8,504 / p <1e-300	42.1 EB05 37.6 · IC025 +5.0 3/3 methods agree	Lab value / disease marker	serious PRR 42.4 · 86% consumer	no trial data cached
Product dose omission issue	138	10.0 8.6–11.7ROR 11.4 · χ² 1,123 / p 1e-246	9.9 EB05 8.5 · IC025 +3.0 3/3 methods agree	Reporting / coding artifact	serious PRR 30.8 · 88% consumer	no trial data cached
Product dose omission in error	37	28.4 20.7–39.0ROR 29.5 · χ² 952 / p 2e-209	28.0 EB05 21.2 · IC025 +3.8 3/3 methods agree	Reporting / coding artifact	serious PRR 21.4 · 100% consumer	no trial data cached
Chromaturia	16	14.6 9.0–23.8ROR 14.8 · χ² 190 / p 2e-43	13.8 EB05 8.3 · IC025 +2.5 3/3 methods agree	Not in label — unassessed	–	no trial data cached
Jaundice (yellowing of skin/eyes)	15	11.5 6.9–19.0ROR 11.6 · χ² 133 / p 4e-31	10.2 EB05 6.0 · IC025 +2.2 3/3 methods agree	Labeled — boxed warning exact term	serious PRR 5.7	no trial data cached
Brain fog	13	18.4 10.7–31.6ROR 18.6 · χ² 197 / p 6e-45	17.5 EB05 10.3 · IC025 +2.5 3/3 methods agree	Not in label — unassessed	serious PRR 9.6	no trial data cached
Laboratory test abnormal	12	8.4 4.8–14.7ROR 8.5 · χ² 71 / p 2e-17	6.8 EB05 4.0 · IC025 +1.7 3/3 methods agree	Reporting / coding artifact	–	no trial data cached
Impaired work ability	12	11.5 6.6–20.3ROR 11.7 · χ² 105 / p 5e-25	9.8 EB05 5.4 · IC025 +2.0 3/3 methods agree	Not in label — unassessed	serious PRR 4.3	no trial data cached
Thirst	7	8.1 3.9–16.9ROR 8.1 · χ² 37 / p 7e-10	5.8 EB05 2.9 · IC025 +1.2 3/3 methods agree	Not in label — unassessed	serious PRR 15.1	no trial data cached
Initial insomnia	7	14.2 6.8–29.6ROR 14.3 · χ² 73 / p 7e-18	11.2 EB05 4.7 · IC025 +1.6 3/3 methods agree	Labeled adverse reaction exact term	–	no trial data cached
Sickle cell anaemia with crisis	5	6.5 2.7–15.6ROR 6.6 · χ² 18 / p 1e-5	4.4 EB05 2.0 · IC025 +0.6 3/3 methods agree	Not in label — unassessed	serious PRR 32.1	no trial data cached
Full blood count abnormal	5	9.8 4.1–23.5ROR 9.8 · χ² 31 / p 1e-8	6.2 EB05 2.6 · IC025 +0.9 3/3 methods agree	Lab value / disease marker	serious PRR 9.2	no trial data cached
Therapy change	4	8.6 3.2–22.8ROR 8.6 · χ² 20 / p 5e-6	5.0 EB05 2.1 · IC025 +0.5 3/3 methods agree	Reporting / coding artifact	serious n=1	no trial data cached
Libido decreased (lower sex drive)	3	5.0 1.6–15.5ROR 5.0 · χ² 6 / p 0.007	3.2 EB05 1.3 · IC025 -0.4 PRR-only: not supported by EBGM or IC	Not in label — unassessed	–	no trial data cached

Unlabeled candidate signals

Every screened signal with n ≥ 20 was either labeled, an indication term, or an artifact pattern — no unlabeled candidate stood out in the top signals.

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 Mitapivat?

In FDA FAERS, the most frequently co-reported reaction terms for Mitapivat are haemoglobin decreased, chromaturia, jaundice, brain fog, impaired work ability, thirst. 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 Mitapivat have an FDA boxed warning?

Yes. The current FDA label carries a boxed warning: HEPATOCELLULAR INJURY AQVESME can cause serious hepatocellular injury. Measure liver laboratory tests (ALT, AST, alkaline phosphatase, and total bilirubin with fractionation) at baseline and every 4 weeks for 24 weeks and then as clinically indicated. Avoid use of AQVESME in patients with cirrhosis.

Do FAERS signals prove that Mitapivat 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 1d4e2654-1636-0d01-e063-6394a90af303, effective 20260827; matched on openfda.generic_name.exact:"MITAPIVAT")
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, 14 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.