What Is Carglumic Acid and How Does It Work?
Carglumic Acid is a Carbamoyl Phosphate Synthetase 1 (CPS 1) activator is indicated as:
- 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
- adjunctive therapy to standard of care for acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA)
What Are Side Effects Associated with Using Carglumic Acid?
Common side effects of Carglumic Acid include:
- vomiting
- diarrhea
- stomach pain
- headache
- stuffy nose
- sore throat
- ear infection
- changes in sense of taste
- fever
- infection
- flu symptoms
- weight loss
- sleepiness
- skin rash
Serious side effects of Carglumic Acid include:
- hives
- difficulty breathing
- swelling of the face, lips, tongue, or throat
- changes in the mental status
- thinking problems
- drowsiness
- loss of balance
- involuntary eye movements
- severe pain in the upper stomach spreading to the back
- nausea
- vomiting
- headache
- hunger
- sweating
- irritability
- dizziness
- fast heart rate
- anxiety
- shakiness
- increased thirst or urination
- constipation
- muscle pain or weakness
- leg cramps
- numbness or tingling
- feeling jittery
- irregular heartbeats
- fluttering in the chest
- choking feeling
- fever
- ear pain or full feeling
- trouble hearing
- drainage from the ear
- fussiness in a child
- pale skin
- unusual tiredness
- lightheadedness
- shortness of breath
- cold hands and feet
- mouth sores
- skin sores
- sore throat
- cough
Rare side effects of Carglumic Acid include:
- none
Seek medical care or call 911 at once if you have the following serious side effects:
- Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
- Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
- Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.
This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.
What Are the Dosages of Carglumic Acid?
Adult and pediatric dosage
Dispersible tablet
- 200 mg
Hyperammonemia due to NAGS deficiency
Adult and pediatric dosage
- Acute hyperammonemia
- 100-250 mg/kg/day orally divided into 2-4 doses: round to the nearest 100 mg (ie, half of a tablet)
- Chronic hyperammonemia
- 10-100 mg/kg/day orally/nasogastric tube divided into 2-4 doses; round to the nearest 100 mg
- Hyperammonaemia due to PA or MMA
- Adult dosage
- 3.3 g/m2/day orally divided into 2 equal doses (administered 12 hours apart) and rounded up to the next multiple of 50 mg (ie, 1/4 of a tablet)
- Continue treatment until ammonia level is less than 50 micromol/L and for a maximum duration of 7 days
- Pediatric dosage
- Children less than or equal to15 kg: 150 mg/kg/day orally
- Children above15 kg: 3.3 g/m2/day orally
- Divided into 2 equal doses (administered 12 hours apart) and rounded up to the next multiple of 50 mg (ie, 1/4 of a tablet)
- Continue treatment until ammonia level is less than 50 micromol/L and for a maximum duration of 7 days
Dosage Considerations – Should be Given as Follows:
- See “Dosages”
What Other Drugs Interact with Carglumic Acid?
If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.
- Carglumic Acid has no noted severe interactions with any other drugs
- Carglumic Acid has no noted serious interactions with any other drugs
- Carglumic Acid has no noted moderate interactions with any other drugs
- Carglumic Acid has no noted minor interactions with any other drugs
This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.
What Are Warnings and Precautions for Carglumic Acid?
Contraindications
- None
Effects of drug abuse
- None
Short-Term Effects
- See “What Are Side Effects Associated with Using Carglumic Acid?”
Long-Term Effects
- See “What Are Side Effects Associated with Using Carglumic Acid?”
Cautions
- Plasma concentrations of Carglumic Acid increased in patients with renal impairment; reduce the dosage in patients with moderate or severe renal impairment; the pharmacokinetics of Carglumic Acid have not been evaluated in patients with end-stage renal disease
- Hyperammonemia
- Monitor serum ammonia levels and adjust the dose to maintain within the normal range for age
- Treat acute hyperammonemia episodes as a life-threatening emergency
- Treatment of severe hyperammonemia may require dialysis, preferably hemodialysis and/or hemofiltration, to reduce plasma ammonia concentration
- Untreated hyperammonemia can result in brain damage and death, and prompt use of all therapies necessary to reduce plasma ammonia levels is essential
- Protein restriction during an acute hyperammonemic episode is recommended for no longer than 12-36 hours while maximizing caloric supplementation to reverse catabolism; protein should be reintroduced as early as possible, following improvement of metabolic and clinical abnormalities in this setting; during long-term management, dietary protein restriction should be instituted to maintain blood ammonia level within an acceptable range for age
- Ongoing monitoring of plasma ammonia level, neurological status, growth parameters, protein intake/nutritional status (both during acute hyperammonemic episodes and long-term), and relevant laboratory tests in patients receiving therapy should be part of evaluating clinical response to treatment
Pregnancy and Lactation
- Rare case reports of use in pregnant women are insufficient to inform a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes
- Pregnancy pharmacovigilance program for Carglumic Acid exposures: 1-888-575-8344
- Pregnant women with urea cycle disorders may experience an increase in catabolic stress which can trigger a hyperammonemic crisis both in intrapartum and in postpartum (3-14 days post-partum) periods
- Maternal complications related to hyperammonemia crisis can include neurological impairment, coma, and in some cases death
- Lactation
- Unknown whether a drug is excreted in human milk
- Present in rat milk and an increase in mortality and impairment of body weight gain occurred in neonatal rats nursed by mothers receiving Carglumic Acid
- Breastfeeding is not recommended during therapy; treatment is continuous and life-long for NAGS deficiency patients
