What the label lists and what reports show
Carglumic acid side effects
Carglumic acid's FDA label: “Most common adverse reactions (≥13%) are vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache.” FAERS holds 745 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 vomiting (147 reports).
- Generic listed
Check it at the source: the carglumic acid FDA label on DailyMed (effective January 25, 2024, Recordati Rare Diseases); the FDA FAERS public dashboard.
What does the label list?
From the label: adverse reactionsMost common adverse reactions (≥13%) are vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache.
Table 1: Adverse Reactions Reported in ≥ 2 Patients with NAGS deficiency Treated with CARBAGLU in the Retrospective Case Series
| Adverse Reaction | Number of Patients (N) (%) |
|---|---|
| Vomiting | 6 (26) |
| Abdominal pain | 4 (17) |
| Pyrexia | 4 (17) |
| Tonsillitis | 4 (17) |
| Anemia | 3 (13) |
| Diarrhea | 3 (13) |
| Ear infection | 3 (13) |
| Infections | 3 (13) |
| Nasopharyngitis | 3 (13) |
| Hemoglobin decreased | 3 (13) |
| Headache | 3 (13) |
| Dysgeusia | 2 (9) |
| Asthenia | 2 (9) |
| Hyperhidrosis | 2 (9) |
| Influenza | 2 (9) |
| Pneumonia | 2 (9) |
| Weight decreased | 2 (9) |
| Anorexia | 2 (9) |
| Somnolence | 2 (9) |
| Rash | 2 (9) |
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, continuedADVERSE REACTIONS NAGS deficiency: Most common adverse reactions (≥13%) are vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache. ( 6.1 ) PA and MMA : Most common adverse reactions (≥5%) are neutropenia, anemia, vomiting, electrolyte imbalance, decreased appetite, hypoglycemia, lethargy/stupor, encephalopathy and pancreatitis/lipase increased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, 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. Acute and Chronic Hyperammonemia due to NAGS Deficiency In a retrospective case series of 23 NAGS deficiency patients treated with CARBAGLU, 17 of the 23 patients reported an adverse reaction. The most common adverse reactions (occurring in ≥ 13% of patients) were vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache. Table 1 summarizes adverse reactions occurring in 2 or more patients treated with CARBAGLU.
Source: FDA drug label, Recordati Rare Diseases, effective January 25, 2024 (SPL set 10175e73-5172-4dde-a508-8a88b7afc0a1), via openFDA; the full label on DailyMed
What do FAERS reports show?
745 reports list carglumic acid among the medicines taken, each a report of something that happened, not proof the medicine caused it; 151 are coded as a death and 401 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 19 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?
- Carglumic acid: 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