Patisiran side effects: what 2,810 FDA adverse event reports show
Drug class
Not stated in label
Brand names
Onpattro
Who reports
2,810 reports · 68% serious · 418 with death outcome · 34% female
Used for
ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ONPATTRO contains a transthyretin-directed small interfering RNA and is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
What it is
ONPATTRO contains patisiran, a double-stranded small interfering ribonucleic acid (siRNA), formulated as a lipid complex for delivery to hepatocytes. Patisiran specifically binds to a genetically conserved sequence in the 3' untranslated region (3'UTR) of mutant and wild-type transthyretin (TTR) messenger RNA (mRNA).
Boxed warning
None in the current label
Causality screen of the 40 strongest signals
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.
- Outcome field, not a reaction Death is a report outcome, not a reaction term. It is dominated by the underlying illness; see the seriousness profile and the boxed warning instead.
- 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. 20 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Intentional dose omission | 114 | 31.2 26.1–37.4ROR 32.5 · χ² 3,296 / p <1e-300 | 31.0 EB05 26.5 · IC025 +4.5 3/3 methods agree | Reporting / coding artifact | serious PRR 43.0 | no trial data cached |
| Infusion related reaction (reaction during infusion) | 129 | 14.6 12.3–17.3ROR 15.3 · χ² 1,623 / p <1e-300 | 14.5 EB05 12.5 · IC025 +3.5 3/3 methods agree | Labeled (related wording) related wording | serious PRR 4.7 | no trial data cached |
| Hospice care | 15 | 11.4 6.9–18.9ROR 11.4 · χ² 132 / p 9e-31 | 10.0 EB05 6.0 · IC025 +2.2 3/3 methods agree | Not in label — unassessed | serious PRR 13.1 | no trial data cached |
| Poor venous access | 15 | 10.9 6.6–18.0ROR 10.9 · χ² 124 / p 3e-29 | 9.5 EB05 5.7 · IC025 +2.2 3/3 methods agree | Not in label — unassessed | serious n=8 | no trial data cached |
| Product preparation error | 29 | 10.5 7.3–15.0ROR 10.6 · χ² 239 / p 4e-54 | 9.8 EB05 6.9 · IC025 +2.6 3/3 methods agree | Reporting / coding artifact | serious n=2 | no trial data cached |
| Cardiac failure (heart failure) | 100 | 9.3 7.7–11.3ROR 9.6 · χ² 734 / p 6e-162 | 9.1 EB05 7.6 · IC025 +2.8 3/3 methods agree | Not in label — unassessed | serious PRR 7.9 | no trial data cached |
| Therapy change | 12 | 9.3 5.3–16.4ROR 9.3 · χ² 81 / p 1e-19 | 7.6 EB05 4.4 · IC025 +1.8 3/3 methods agree | Reporting / coding artifact | serious n=9 | no trial data cached |
| Carpal tunnel syndrome | 16 | 8.5 5.2–13.9ROR 8.6 · χ² 99 / p 2e-23 | 7.3 EB05 4.6 · IC025 +2.0 3/3 methods agree | Not in label — unassessed | serious PRR 7.3 | no trial data cached |
| Therapy interrupted | 76 | 8.2 6.6–10.3ROR 8.4 · χ² 476 / p 6e-106 | 7.9 EB05 6.5 · IC025 +2.6 3/3 methods agree | Reporting / coding artifact | serious PRR 15.2 | no trial data cached |
| Infusion site pain | 14 | 8.2 4.9–13.8ROR 8.2 · χ² 81 / p 1e-19 | 6.8 EB05 4.2 · IC025 +1.8 3/3 methods agree | Labeled (related wording) related wording | serious n=8 | no trial data cached |
| Insurance issue | 16 | 8.0 4.9–13.1ROR 8.1 · χ² 92 / p 5e-22 | 6.8 EB05 4.4 · IC025 +1.9 3/3 methods agree | Not in label — unassessed | serious PRR 3.5 | no trial data cached |
| Intercepted product preparation error | 13 | 8.0 4.6–13.7ROR 8.0 · χ² 72 / p 9e-18 | 6.6 EB05 4.0 · IC025 +1.7 3/3 methods agree | Not in label — unassessed | – | no trial data cached |
| Hospitalisation | 132 | 6.5 5.5–7.6ROR 6.7 · χ² 609 / p 1e-134 | 6.3 EB05 5.5 · IC025 +2.4 3/3 methods agree | Reporting / coding artifact | serious PRR 5.6 | no trial data cached |
| Polyneuropathy | 10 | 6.3 3.4–11.7ROR 6.3 · χ² 39 / p 2e-10 | 5.0 EB05 2.9 · IC025 +1.2 3/3 methods agree | Likely indication confounding term in indications; also in warnings | serious PRR 3.2 | no trial data cached |
| Generalised oedema | 10 | 6.1 3.3–11.3ROR 6.1 · χ² 37 / p 5e-10 | 4.9 EB05 2.8 · IC025 +1.2 3/3 methods agree | Not in label — unassessed | serious PRR 5.7 | no trial data cached |
| Product administration error | 16 | 6.0 3.7–9.8ROR 6.0 · χ² 62 / p 2e-15 | 5.2 EB05 3.4 · IC025 +1.5 3/3 methods agree | Reporting / coding artifact | serious PRR 4.7 | no trial data cached |
| Fluid overload | 11 | 5.6 3.1–10.0ROR 5.6 · χ² 37 / p 7e-10 | 4.6 EB05 2.8 · IC025 +1.2 3/3 methods agree | Not in label — unassessed | serious PRR 5.6 | no trial data cached |
| Localised infection | 18 | 5.6 3.5–8.8ROR 5.6 · χ² 63 / p 1e-15 | 4.9 EB05 3.3 · IC025 +1.5 3/3 methods agree | Labeled (related wording) related wording | serious PRR 5.8 | no trial data cached |
| Neuralgia | 18 | 5.5 3.5–8.8ROR 5.6 · χ² 63 / p 1e-15 | 4.9 EB05 3.3 · IC025 +1.5 3/3 methods agree | Not in label — unassessed | serious PRR 3.9 | no trial data cached |
| Ischaemic stroke | 12 | 5.0 2.8–8.8ROR 5.0 · χ² 35 / p 2e-9 | 4.3 EB05 2.6 · IC025 +1.1 3/3 methods agree | Not in label — unassessed | serious PRR 4.3 | no trial data cached |
| Product dose omission | 44 | 5.0 3.7–6.7ROR 5.0 · χ² 136 / p 1e-31 | 4.7 EB05 3.7 · IC025 +1.7 3/3 methods agree | Reporting / coding artifact | serious PRR 6.3 | no trial data cached |
| Fluid retention | 33 | 4.8 3.5–6.8ROR 4.9 · χ² 97 / p 3e-23 | 4.6 EB05 3.4 · IC025 +1.6 3/3 methods agree | Not in label — unassessed | serious PRR 5.0 | no trial data cached |
| Unevaluable event | 48 | 4.7 3.6–6.3ROR 4.8 · χ² 138 / p 3e-32 | 4.5 EB05 3.6 · IC025 +1.7 3/3 methods agree | Reporting / coding artifact | serious PRR 4.0 · 71% consumer | no trial data cached |
| Ill-defined disorder | 38 | 4.7 3.4–6.4ROR 4.7 · χ² 106 / p 4e-25 | 4.4 EB05 3.4 · IC025 +1.6 3/3 methods agree | Not in label — unassessed | serious PRR 3.9 | no trial data cached |
| Product dose omission issue | 176 | 4.6 4.0–5.3ROR 4.9 · χ² 501 / p 3e-111 | 4.6 EB05 4.0 · IC025 +1.9 3/3 methods agree | Reporting / coding artifact | serious PRR 11.2 | no trial data cached |
| Blood pressure decreased | 40 | 4.5 3.3–6.1ROR 4.6 · χ² 106 / p 4e-25 | 4.3 EB05 3.3 · IC025 +1.6 3/3 methods agree | Labeled (related wording) related wording | serious PRR 3.8 | no trial data cached |
| Medication error | 33 | 4.4 3.2–6.2ROR 4.5 · χ² 85 / p 2e-20 | 4.2 EB05 3.1 · IC025 +1.5 3/3 methods agree | Reporting / coding artifact | serious PRR 2.4 | no trial data cached |
| Orthostatic hypotension (dizziness on standing) | 10 | 4.3 2.3–7.9ROR 4.3 · χ² 22 / p 1e-6 | 3.7 EB05 2.2 · IC025 +0.8 3/3 methods agree | Not in label — unassessed | serious PRR 3.7 | no trial data cached |
| Dysstasia | 16 | 4.1 2.5–6.6ROR 4.1 · χ² 34 / p 3e-9 | 3.7 EB05 2.4 · IC025 +1.1 3/3 methods agree | Not in label — unassessed | serious PRR 3.9 | no trial data cached |
| Atrial fibrillation | 51 | 3.9 3.0–5.1ROR 4.0 · χ² 108 / p 2e-25 | 3.8 EB05 3.0 · IC025 +1.5 3/3 methods agree | Not in label — unassessed | serious PRR 3.4 | no trial data cached |
| Neuropathy peripheral (nerve damage in hands/feet) | 49 | 3.9 2.9–5.1ROR 3.9 · χ² 102 / p 3e-24 | 3.7 EB05 2.9 · IC025 +1.4 3/3 methods agree | Labeled (related wording) related wording | serious PRR 2.5 | no trial data cached |
| Heart rate decreased | 17 | 3.6 2.2–5.8ROR 3.6 · χ² 29 / p 3e-8 | 3.3 EB05 2.2 · IC025 +0.9 3/3 methods agree | Labeled (related wording) related wording | serious PRR 2.9 | no trial data cached |
| Inappropriate schedule of product administration | 84 | 3.5 2.9–4.4ROR 3.6 · χ² 152 / p 3e-35 | 3.5 EB05 2.9 · IC025 +1.4 3/3 methods agree | Reporting / coding artifact | serious PRR 6.3 | no trial data cached |
| Balance disorder | 41 | 3.5 2.6–4.8ROR 3.6 · χ² 72 / p 9e-18 | 3.4 EB05 2.6 · IC025 +1.3 3/3 methods agree | Not in label — unassessed | serious PRR 3.3 | no trial data cached |
| Cardiac disorder | 39 | 3.1 2.3–4.2ROR 3.1 · χ² 54 / p 1e-13 | 3.0 EB05 2.3 · IC025 +1.1 3/3 methods agree | Not in label — unassessed | serious PRR 2.7 | no trial data cached |
| Disease progression | 45 | 2.8 2.1–3.8ROR 2.9 · χ² 52 / p 2e-13 | 2.8 EB05 2.2 · IC025 +1.0 3/3 methods agree | Reporting / coding artifact | serious PRR 2.4 | no trial data cached |
| Death | 269 | 2.3 2.1–2.6ROR 2.5 · χ² 212 / p 3e-48 | 2.3 EB05 2.1 · IC025 +1.0 3/3 methods agree | Outcome field, not a reaction | serious PRR 2.0 | no trial data cached |
| Blindness | 12 | 2.2 1.3–3.9ROR 2.2 · χ² 7 / p 0.005 | 2.1 EB05 1.3 · IC025 +0.1 | Labeled adverse reaction exact term | serious PRR 1.9 | no trial data cached |
| Cerebrovascular accident (stroke) | 50 | 2.2 1.7–2.9ROR 2.2 · χ² 32 / p 1e-8 | 2.2 EB05 1.7 · IC025 +0.6 | Not in label — unassessed | serious PRR 1.9 | no trial data cached |
| No adverse event | 51 | 2.1 1.6–2.8ROR 2.2 · χ² 30 / p 2e-8 | 2.1 EB05 1.7 · IC025 +0.6 | Reporting / coding artifact | serious PRR 4.1 · 90% clinician | no trial data cached |
Trial evidence (ClinicalTrials.gov)
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 |
|---|---|---|---|---|---|---|
| Death | 269 | 2.3 2.1–2.6ROR 2.5 · χ² 212 / p 3e-48 | 2.3 EB05 2.1 · IC025 +1.0 3/3 methods agree | Outcome field, not a reaction | serious PRR 2.0 | no trial data cached |
| Product dose omission issue | 176 | 4.6 4.0–5.3ROR 4.9 · χ² 501 / p 3e-111 | 4.6 EB05 4.0 · IC025 +1.9 3/3 methods agree | Reporting / coding artifact | serious PRR 11.2 | no trial data cached |
| Hospitalisation | 132 | 6.5 5.5–7.6ROR 6.7 · χ² 609 / p 1e-134 | 6.3 EB05 5.5 · IC025 +2.4 3/3 methods agree | Reporting / coding artifact | serious PRR 5.6 | no trial data cached |
| Infusion related reaction (reaction during infusion) | 129 | 14.6 12.3–17.3ROR 15.3 · χ² 1,623 / p <1e-300 | 14.5 EB05 12.5 · IC025 +3.5 3/3 methods agree | Labeled (related wording) related wording | serious PRR 4.7 | no trial data cached |
| Intentional dose omission | 114 | 31.2 26.1–37.4ROR 32.5 · χ² 3,296 / p <1e-300 | 31.0 EB05 26.5 · IC025 +4.5 3/3 methods agree | Reporting / coding artifact | serious PRR 43.0 | no trial data cached |
| Cardiac failure (heart failure) | 100 | 9.3 7.7–11.3ROR 9.6 · χ² 734 / p 6e-162 | 9.1 EB05 7.6 · IC025 +2.8 3/3 methods agree | Not in label — unassessed | serious PRR 7.9 | no trial data cached |
| Inappropriate schedule of product administration | 84 | 3.5 2.9–4.4ROR 3.6 · χ² 152 / p 3e-35 | 3.5 EB05 2.9 · IC025 +1.4 3/3 methods agree | Reporting / coding artifact | serious PRR 6.3 | no trial data cached |
| Therapy interrupted | 76 | 8.2 6.6–10.3ROR 8.4 · χ² 476 / p 6e-106 | 7.9 EB05 6.5 · IC025 +2.6 3/3 methods agree | Reporting / coding artifact | serious PRR 15.2 | no trial data cached |
| No adverse event | 51 | 2.1 1.6–2.8ROR 2.2 · χ² 30 / p 2e-8 | 2.1 EB05 1.7 · IC025 +0.6 | Reporting / coding artifact | serious PRR 4.1 · 90% clinician | no trial data cached |
| Atrial fibrillation | 51 | 3.9 3.0–5.1ROR 4.0 · χ² 108 / p 2e-25 | 3.8 EB05 3.0 · IC025 +1.5 3/3 methods agree | Not in label — unassessed | serious PRR 3.4 | no trial data cached |
| Cerebrovascular accident (stroke) | 50 | 2.2 1.7–2.9ROR 2.2 · χ² 32 / p 1e-8 | 2.2 EB05 1.7 · IC025 +0.6 | Not in label — unassessed | serious PRR 1.9 | no trial data cached |
| Neuropathy peripheral (nerve damage in hands/feet) | 49 | 3.9 2.9–5.1ROR 3.9 · χ² 102 / p 3e-24 | 3.7 EB05 2.9 · IC025 +1.4 3/3 methods agree | Labeled (related wording) related wording | serious PRR 2.5 | no trial data cached |
| Unevaluable event | 48 | 4.7 3.6–6.3ROR 4.8 · χ² 138 / p 3e-32 | 4.5 EB05 3.6 · IC025 +1.7 3/3 methods agree | Reporting / coding artifact | serious PRR 4.0 · 71% consumer | no trial data cached |
| Disease progression | 45 | 2.8 2.1–3.8ROR 2.9 · χ² 52 / p 2e-13 | 2.8 EB05 2.2 · IC025 +1.0 3/3 methods agree | Reporting / coding artifact | serious PRR 2.4 | no trial data cached |
| Product dose omission | 44 | 5.0 3.7–6.7ROR 5.0 · χ² 136 / p 1e-31 | 4.7 EB05 3.7 · IC025 +1.7 3/3 methods agree | Reporting / coding artifact | serious PRR 6.3 | 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.
- Cardiac failure — n=100, PRR 9.3
- Fluid retention — n=33, PRR 4.8
- Ill-defined disorder — n=38, PRR 4.7
- Atrial fibrillation — n=51, PRR 3.9
- Balance disorder — n=41, PRR 3.5
- Cardiac disorder — n=39, PRR 3.1
- Cerebrovascular accident — n=50, PRR 2.2
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 Patisiran?
In FDA FAERS, the most frequently co-reported reaction terms for Patisiran are infusion related reaction, cardiac failure, atrial fibrillation, cerebrovascular accident, neuropathy peripheral. 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 Patisiran have an FDA boxed warning?
No. The FDA label retrieved for Patisiran has no boxed warning; see the label's Warnings and Precautions for the recognised risks.
Do FAERS signals prove that Patisiran 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 e87ec36f-b4b4-49d4-aea4-d4ffb09b0970, effective 20250908; matched on
openfda.generic_name.exact:"PATISIRAN") - 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, fieldpatient.drug.openfda.generic_name.exact, 60 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.