Cerliponase Alfa side effects: what 526 FDA adverse event reports show

Cerliponase Alfa · Brineura · 15 reaction terms · 239 drug–reaction co-reports · data 2026-09-14

Drug class

Hydrolytic lysosomal n-terminal tripeptidyl peptidase

FDA label, Indications wording

Brand names

Brineura

openFDA label

Who reports

526 reports · 72% serious · 24 with death outcome · 45% female

openFDA FAERS counts, all years, deduplication not applied

Used for

BRINEURA is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. BRINEURA is a hydrolytic lysosomal N-terminal tripeptidyl pep.

FDA label, Indications and Usage

What it is

Cerliponase alfa is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line. The active substance is a recombinant human tripeptidyl peptidase-1 (rhTPP1), a lysosomal exopeptidase.

FDA label, Description section

Boxed warning

HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

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 13 strongest signals

Labeled adverse reaction 4Labeled (related wording) 1Reporting / coding artifact 6Not in label — unassessed 2

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.
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.
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
Device related infection	30	77.6 54.8–110.0ROR 82.3 · χ² 2,191 / p <1e-300	74.0 EB05 54.3 · IC025 +4.5 3/3 methods agree	Reporting / coding artifact	serious PRR 63.4	no trial data cached
Device occlusion	16	45.9 28.3–74.4ROR 47.3 · χ² 658 / p 2e-145	43.6 EB05 28.5 · IC025 +3.4 3/3 methods agree	Reporting / coding artifact	serious n=13	no trial data cached
Myoclonus	13	43.5 25.4–74.4ROR 44.6 · χ² 498 / p 1e-110	41.1 EB05 25.5 · IC025 +3.1 3/3 methods agree	Not in label — unassessed	serious PRR 36.6	no trial data cached
Dystonia	15	30.7 18.7–50.6ROR 31.6 · χ² 402 / p 8e-90	29.7 EB05 19.1 · IC025 +3.1 3/3 methods agree	Not in label — unassessed	serious PRR 16.2	no trial data cached
Device leakage	28	26.4 18.4–37.8ROR 27.8 · χ² 660 / p 1e-145	26.0 EB05 18.8 · IC025 +3.6 3/3 methods agree	Reporting / coding artifact	serious n=11 · 82% consumer	no trial data cached
Seizure	75	26.1 21.1–32.1ROR 30.2 · χ² 1,792 / p <1e-300	25.9 EB05 21.3 · IC025 +4.1 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 18.7 · 76% consumer	no trial data cached
Status epilepticus	7	24.7 11.8–51.5ROR 25.0 · χ² 136 / p 9e-32	22.5 EB05 10.3 · IC025 +2.0 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 19.7	no trial data cached
Cyanosis (blue skin from low oxygen)	7	17.7 8.5–37.0ROR 17.9 · χ² 94 / p 1e-22	15.1 EB05 6.2 · IC025 +1.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 15.0	no trial data cached
Generalised tonic-clonic seizure	7	17.1 8.2–35.6ROR 17.3 · χ² 90 / p 1e-21	14.4 EB05 5.9 · IC025 +1.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 13.8	no trial data cached
Needle issue	8	11.2 5.6–22.2ROR 11.3 · χ² 64 / p 6e-16	8.5 EB05 4.1 · IC025 +1.6 3/3 methods agree	Labeled adverse reaction exact term	serious n=1	no trial data cached
Device dislocation	8	7.3 3.7–14.6ROR 7.4 · χ² 38 / p 4e-10	5.5 EB05 2.9 · IC025 +1.2 3/3 methods agree	Reporting / coding artifact	serious PRR 3.8	no trial data cached
Device breakage	6	6.1 2.7–13.5ROR 6.1 · χ² 21 / p 3e-6	4.4 EB05 2.2 · IC025 +0.7 3/3 methods agree	Reporting / coding artifact	serious PRR 10.2	no trial data cached
Device issue	11	5.0 2.8–9.0ROR 5.1 · χ² 32 / p 9e-9	4.2 EB05 2.5 · IC025 +1.1 3/3 methods agree	Reporting / coding artifact	serious PRR 12.1	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
Seizure	75	26.1 21.1–32.1ROR 30.2 · χ² 1,792 / p <1e-300	25.9 EB05 21.3 · IC025 +4.1 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 18.7 · 76% consumer	no trial data cached
Device related infection	30	77.6 54.8–110.0ROR 82.3 · χ² 2,191 / p <1e-300	74.0 EB05 54.3 · IC025 +4.5 3/3 methods agree	Reporting / coding artifact	serious PRR 63.4	no trial data cached
Device leakage	28	26.4 18.4–37.8ROR 27.8 · χ² 660 / p 1e-145	26.0 EB05 18.8 · IC025 +3.6 3/3 methods agree	Reporting / coding artifact	serious n=11 · 82% consumer	no trial data cached
Device occlusion	16	45.9 28.3–74.4ROR 47.3 · χ² 658 / p 2e-145	43.6 EB05 28.5 · IC025 +3.4 3/3 methods agree	Reporting / coding artifact	serious n=13	no trial data cached
Dystonia	15	30.7 18.7–50.6ROR 31.6 · χ² 402 / p 8e-90	29.7 EB05 19.1 · IC025 +3.1 3/3 methods agree	Not in label — unassessed	serious PRR 16.2	no trial data cached
Myoclonus	13	43.5 25.4–74.4ROR 44.6 · χ² 498 / p 1e-110	41.1 EB05 25.5 · IC025 +3.1 3/3 methods agree	Not in label — unassessed	serious PRR 36.6	no trial data cached
Device issue	11	5.0 2.8–9.0ROR 5.1 · χ² 32 / p 9e-9	4.2 EB05 2.5 · IC025 +1.1 3/3 methods agree	Reporting / coding artifact	serious PRR 12.1	no trial data cached
Device dislocation	8	7.3 3.7–14.6ROR 7.4 · χ² 38 / p 4e-10	5.5 EB05 2.9 · IC025 +1.2 3/3 methods agree	Reporting / coding artifact	serious PRR 3.8	no trial data cached
Needle issue	8	11.2 5.6–22.2ROR 11.3 · χ² 64 / p 6e-16	8.5 EB05 4.1 · IC025 +1.6 3/3 methods agree	Labeled adverse reaction exact term	serious n=1	no trial data cached
Generalised tonic-clonic seizure	7	17.1 8.2–35.6ROR 17.3 · χ² 90 / p 1e-21	14.4 EB05 5.9 · IC025 +1.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 13.8	no trial data cached
Cyanosis (blue skin from low oxygen)	7	17.7 8.5–37.0ROR 17.9 · χ² 94 / p 1e-22	15.1 EB05 6.2 · IC025 +1.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 15.0	no trial data cached
Status epilepticus	7	24.7 11.8–51.5ROR 25.0 · χ² 136 / p 9e-32	22.5 EB05 10.3 · IC025 +2.0 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 19.7	no trial data cached
Device breakage	6	6.1 2.7–13.5ROR 6.1 · χ² 21 / p 3e-6	4.4 EB05 2.2 · IC025 +0.7 3/3 methods agree	Reporting / coding artifact	serious PRR 10.2	no trial data cached
Complication associated with device	4	12.1 4.5–32.0ROR 12.1 · χ² 30 / p 2e-8	7.1 EB05 2.5 · IC025 +0.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 7.9	no trial data cached
Hypotonia	4	15.9 6.0–42.2ROR 16.0 · χ² 42 / p 5e-11	10.1 EB05 3.1 · IC025 +0.8 3/3 methods agree	Not in label — unassessed	serious PRR 14.5	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.

✓ A hand-written Bradford Hill causality assessment exists for this drug: Erythropoiesis-stimulating agents (darbepoetin alfa) ↔ Mortality / cardiovascular risk.

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 Cerliponase Alfa?

In FDA FAERS, the most frequently co-reported reaction terms for Cerliponase Alfa are seizure, dystonia, myoclonus, needle issue, generalised tonic-clonic seizure, cyanosis. 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 Cerliponase Alfa have an FDA boxed warning?

Yes. The current FDA label carries a boxed warning: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Do FAERS signals prove that Cerliponase Alfa 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 485ab3c5-0359-4878-872f-2fe1e7df4047, effective 20240805; matched on openfda.generic_name.exact:"CERLIPONASE ALFA")
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, 15 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.