What the label lists and what reports show
Cerliponase alfa side effects
Cerliponase alfa's FDA label: “Most common adverse reactions (≥8%) are: pyrexia, ECG abnormalities, decreased CSF protein, vomiting, seizures, device-related complications, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infections …” FAERS holds 526 reports naming it through July 30, 2026; the reaction reported most often, leaving out the conditions it is used for, product-use terms and outcomes, is pyrexia (90 reports).
- No generic listed
- Boxed warning
Check it at the source: the cerliponase alfa FDA label on DailyMed (effective August 5, 2024, BioMarin Pharmaceutical Inc.); the FDA FAERS public dashboard.
What does the label list?
From the label: adverse reactionsMost common adverse reactions (≥8%) are: pyrexia, ECG abnormalities, decreased CSF protein, vomiting, seizures, device-related complications, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infections, bradycardia, feeling jittery, and hypotension.
Table 2: Adverse Reactions Reported in ≥ 8% of Symptomatic Pediatric Patients with CLN2 Disease in BRINEURA Trial 1 and Trial 2 at Week 96
| Adverse Reaction | Patients Treated with BRINEURA n=24 (%) |
|---|---|
| Increased body temperature Increased body temperature includes: increased body… | 17 (71) |
| ECG abnormalities ECG abnormalities include: non-specific repolarization abnormality,… | 17 (71) |
| Decreased CSF protein | 17 (71) |
| Vomiting | 15 (63) |
| Seizures Seizures include: atonic, generalized tonic-clonic, focal, and absence | 12 (50) |
| Device-related complications Device-related complications include device-related… | 12 (50) |
| Hypersensitivity Hypersensitivity includes only hypersensitivity | 9 (38) |
| Increased CSF protein | 5 (21) |
| Hematoma | 5 (21) |
| Headache | 4 (17) |
| Irritability | 4 (17) |
| Pleocytosis | 4 (17) |
| Device-related infections Device-related infections include: Propionibacterium acnes and… | 2 (8) |
| Bradycardia | 2 (8) |
| Feeling jittery | 2 (8) |
| Hypotension | 2 (8) |
Rows and numbers as the label prints them, under its own column headings (the first rows of a longer table where it runs on). A reaction in a trial table is not necessarily caused by the medicine, and trials differ from everyday use.
From the label: adverse reactions, continuedThe following adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Meningitis and Other Intraventricular Access Device-Related Infections [see Warnings and Precautions (5.2) ] Intraventricular Access Device-Related Complications [see Warnings and Precautions (5.3) ] Cardiovascular Adverse Reactions [see Warnings and Precautions (5.4) ] Infusion-Associated Reactions [see Warnings and Precautions (5.5) ] Most common adverse reactions (≥8%) are: pyrexia, ECG abnormalities, decreased CSF protein, vomiting, seizures, device-related complications, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infections, bradycardia, feeling jittery, and hypotension. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact BioMarin at 1-866-906-6100 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. …
Source: FDA drug label, BioMarin Pharmaceutical Inc., effective August 5, 2024 (SPL set 485ab3c5-0359-4878-872f-2fe1e7df4047), via openFDA; the full label on DailyMed
What do FAERS reports show?
526 reports list cerliponase alfa among the medicines taken, each a report of something that happened, not proof the medicine caused it; 24 are coded as a death and 193 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is. Of the 21 reactions below, 8 are named by the same term (in US spelling) in the label's adverse reactions, warnings or patient leaflet; a reaction the label does not name may still be there under another word, and a report does not show that the medicine caused it.
Reports received each year
The 25 most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026; reactions as MedDRA preferred terms
What next?
- Cerliponase alfa: the full record
- label, generics, price, shortages, recalls
- Report a side effect to the FDA
- MedWatch
- Side effects hub
- the most reported medicines and reactions