Last reviewed 2026-04-20 · How we verify

Carbaglu

At a glance

Approved indications

• Hyperammonemia associated with N-acetylglutamate synthase deficiency

Common side effects

• Vomiting
• Neutropenia
• Abdominal pain
• Pyrexia
• Tonsillitis
• Anemia
• Diarrhea
• Ear infection
• Infections
• Nasopharyngitis
• Hemoglobin decreased
• Headache

Serious adverse events

• Encephalopathy
• Pancreatitis/Lipase increased
• Hypoglycemia
• Lethargy/Stupor
• Cardiomyopathy
• Alanine aminotransferase increased
• Mania
• Pruritus
• Rash
• Rash erythematous

Key clinical trials

• An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
• Long-term Efficacy of Carglumic Acid in Organic Acidemia. (NA)
• Orphan Europe Carbaglu® Surveillance Protocol
• Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
• Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia (PHASE2)
• N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia (PHASE2, PHASE3)
• Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia (PHASE3)
• Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia (PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Carbaglu CI brief — competitive landscape report
• Carbaglu updates RSS · CI watch RSS
• Mendel Tuchman portfolio CI