Carbaglu

Generic name: carglumic acid
Drug class: Miscellaneous uncategorized agents

Medically reviewed by  A Ras MD.

What is Carbaglu?

Carbaglu is used to treat certain pediatric and adult patients with high ammonia levels. Carbaglu is used as Add-on (adjunctive) therapy to standard of care for the treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency ,Maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency, Add-on therapy to standard of care for the treatment of acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA).

Description

CARBAGLU tablets for oral suspension contain 200 mg of carglumic acid. Carglumic acid, the active substance, is a carbamoyl phosphate synthetase 1 (CPS 1) activator and is soluble in boiling water, slightly soluble in cold water, and practically insoluble in organic solvents.

Chemically, carglumic acid is N-carbamoyl-L-glutamic acid or (2S)-2-(carbamoylamino) pentanedioic acid, with a molecular weight of 190.16.

The structural formula is:

Molecular Formula: C6H10N2O5

The inactive ingredients of CARBAGLU are croscarmellose sodium, hypromellose, microcrystalline cellulose, silica colloidal anhydrous, sodium lauryl sulfate, and sodium stearyl fumarate.

Mechanism of Action

Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG) which is produced from glutamate and acetyl-CoA in a reaction catalyzed by N‑acetylglutamate synthase (NAGS), a mitochondrial liver enzyme. NAG acts as the essential allosteric activator of carbamoyl phosphate synthetase 1 (CPS 1), a mitochondrial liver enzyme which catalyzes the first reaction of the urea cycle. The urea cycle, whose role is the disposition of ammonia, includes a series of biochemical reactions in the liver resulting in the conversion of ammonia into urea, which is then excreted through the urine. Carglumic acid acts as a CPS1 activator, improves or restores the function of the urea cycle, and facilitates ammonia detoxification and urea production.

How should I take Carbaglu?

• Read the instructions for use below for information about the right way to take Carbaglu.
• Disperse Carbaglu tablets in water. Do not swallow whole or crushed.
• Take Carbaglu immediately before meals or feedings.
• Carbaglu tablets dispersed in water can be administered orally or via a nasogastric tube or gastrostomy tube as described in the Instructions for Use.

What are the possible side effects of Carbaglu?

The most common adverse reactions (occurring in ≥ 13% of patients) in patients with acute and chronic hyperammonemia due to NAGS deficiency are:

• vomiting
• abdominal pain
• pyrexia
• tonsillitis
• anemia
• diarrhea
• ear infection
• infections
• nasopharyngitis
• hemoglobin decreased
• headache

The most common adverse reactions (≥ 5%) during hyperammonemic episodes in patients with acute hyperammonemia due PA or MMA are:

• neutropenia
• anemia
• vomiting
• electrolyte imbalance
• decreased appetite
• hypoglycemia
• lethargy/stupor
• encephalopathy
• pancreatitis/lipase increased

General information about the safe and effective use of Carbaglu

Medicines are sometimes prescribed for purposes other than those listed in Patient Information leaflets. Do not use Carbaglu for a condition for which it was not prescribed. Do not give Carbaglu to other people, even if they have the same symptoms that you have. It may harm them.

You can ask your doctor or pharmacist for information about Carbaglu that is written for health professionals.

How should I store Carbaglu?

• Before opening, store Carbaglu in a refrigerator between 36°F to 46°F (2°C to 8°C) in the container it comes in.
• After opening, store Carbaglu at room temperature between 59°F to 86°F (15°C to 30°C). Do not store Carbaglu in a refrigerator after opening.
  • Keep Carbaglu tablets in a tightly closed container to protect the tablets from moisture.
  • Write the date the Carbaglu tablet container is opened on the container label. Throw away any unused tablets one month after opening the tablet container.
• Do not use Carbaglu tablets after the expiration date on the tablet container.
• Keep Carbaglu and all medicines out of the reach of children.

What are the ingredients in Carbaglu?

Active ingredient: Carglumic acid

Inactive ingredients: Microcrystalline cellulose, sodium lauryl sulfate, hypromelloses, croscarmellose sodium, silicon dioxide and sodium stearyl fumarate.