Carbaglu (carglumic acid)
An approved treatment for Urea Cycle Disorders and Methylmalonic Acidemia.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Carglumic acid wherever you encounter it, plus the key facts at a glance.
- Generic name
- Carglumic acid
- Brand name
- Carbaglu
- Development codes
- N-carbamylglutamate, N-carbamoyl-L-glutamic acid
- Drug class
- Carbamoyl phosphate synthetase 1 (CPS1) activator
- Manufacturer
- Recordati Rare Diseases
- How it's taken
- Oral tablets mixed in water, 2 to 4 times a day, or through a feeding tube
An oral tablet, mixed in water, that lowers dangerously high blood ammonia.
- Carbaglu was approved in 2010 for NAGS deficiency, a urea cycle disorder, both for sudden ammonia spikes and for long-term control.
- Since January 2021 it is also approved, alongside standard hospital care, for sudden high ammonia in propionic acidemia (PA) and methylmalonic acidemia (MMA).
- The Carbaglu label has no boxed warning.
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Where Carglumic acid fits
Carbaglu treats both sudden and long-term high ammonia caused by NAGS deficiency, a urea cycle disorder, by standing in for the missing NAG switch, and generic carglumic acid tablets carry the same NAGS deficiency uses[1][6].
- For other urea cycle disorders, the nitrogen-binding medicines Ravicti (glycerol phenylbutyrate) and Buphenyl (sodium phenylbutyrate) are approved for long-term care alongside a protein-restricted diet, and the Ravicti label says its safety and efficacy in NAGS deficiency have not been established[8][9].
- For propionic acidemia and methylmalonic acidemia, Carbaglu is approved only as an add-on during acute ammonia spikes, on top of standard care such as intravenous glucose, insulin, L-carnitine, protein restriction and dialysis[1].
- The PA and MMA trial compared Carbaglu with placebo and found a median of 1.5 days against 2.0 days to normal ammonia or hospital discharge, a 0.5-day difference whose 95% confidence interval included no difference, and no trial has compared Carbaglu head to head with another ammonia-lowering medicine[1].
- Families can ask their metabolic specialist whether Carbaglu belongs in the child's written emergency plan for high ammonia, and how the dose would be worked out.
How Carglumic acid works
The liver clears ammonia, a toxic waste product of protein, through a chain of reactions called the urea cycle, which turns ammonia into urea that leaves the body in urine. The first enzyme in that chain, carbamoyl phosphate synthetase 1 (CPS1), only works when a helper molecule called N-acetylglutamate (NAG) switches it on[1]. NAG is made by an enzyme called N-acetylglutamate synthase (NAGS), and people with NAGS deficiency cannot make enough of it, which leaves the urea cycle stalled and ammonia rising[1].
Carglumic acid is a man-made copy of NAG's shape that switches CPS1 on in its place, which the label says improves or restores urea cycle function and helps the body clear ammonia[1]. In propionic acidemia and methylmalonic acidemia, Carbaglu is used only during acute ammonia spikes, on top of standard hospital treatment[1].
Side effects and safety
- No warnings section. the Carbaglu label lists no contraindications and has no Warnings and Precautions section
- Kidney function. carglumic acid leaves the body mainly through the kidneys, and the label lowers the dose for moderate or severe kidney impairment
- Very high doses. one patient given 650mg/kg a day had a fast heart rate, heavy sweating, extra airway mucus, raised temperature and restlessness, which went away when the dose was lowered
- Plasma ammonia levels, with the dose adjusted to keep ammonia in the normal range for the patient's age
- Kidney function, since the dose is lower with moderate or severe kidney impairment
The Carbaglu label has no boxed warning, no contraindications and no Warnings and Precautions section[1]. In the NAGS deficiency case series, 17 of 23 patients reported a side effect, and the most common were vomiting (26%), abdominal pain (17%), fever (17%), tonsillitis (17%) and, at 13% each, anemia, diarrhea, ear infection, infections, cold symptoms, low hemoglobin and headache[1].
In the PA and MMA trial, counted per hospital episode, at least 1 side effect was reported in 42% of episodes, and those more common with Carbaglu than placebo included neutropenia, a drop in infection-fighting white blood cells (14% vs 8%), anemia (12% vs 8%), vomiting (7% vs 2%), electrolyte imbalance (7% vs 4%), low blood sugar (5% vs 2%), decreased appetite (5% vs 2%), lethargy or stupor (5% vs 2%), encephalopathy, a brain function problem (5% vs 0), and pancreatitis or raised lipase (5% vs 0)[1].
Since approval, mania, itching and rashes have been reported, without a known rate[1]. One patient given 650mg/kg a day developed a fast heart rate, heavy sweating, extra airway mucus, raised temperature and restlessness, which resolved when the dose came down[1]. For pregnancy, the label says case reports are too few to judge risk, notes that untreated NAGS deficiency, PA and MMA can cause irreversible brain damage and death in pregnant women, and points to a pregnancy exposure registry; it is not known whether carglumic acid passes into human milk[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Carglumic acid
Carbaglu comes as 200mg tablets for oral suspension, scored to split into 4 equal parts[1]. The tablets are stirred into water, at least 2.5mL per whole, half or quarter tablet, and taken right away, just before meals or feedings; they are not swallowed whole or crushed, and the label says mixing in foods or liquids other than water has not been studied and is not recommended[1].
The mixture can be swallowed from a cup, given by oral syringe, or given through a nasogastric or gastrostomy feeding tube[1]. For NAGS deficiency, the label dose is 100mg/kg to 250mg/kg a day for acute high ammonia and 10mg/kg to 100mg/kg a day for maintenance, split into 2 to 4 doses and adjusted to keep ammonia in the normal range for age[1].
For acute high ammonia in PA or MMA, the dose is 150mg/kg a day for patients 15kg or less, or 3.3g per square meter of body surface area a day for patients over 15kg, split into 2 doses 12 hours apart, until ammonia falls below 50µmol/L and for no more than 7 days[1]. Doses are lower for moderate or severe kidney impairment[1]. Unopened bottles go in the refrigerator; once opened, the bottle is kept at room temperature, tightly closed, and thrown away 1 month after opening[1].
Availability and cost
Generic versions may be available at a lower cost. Ask your pharmacist.
The label gives no price. Recordati offers a $0 copay for qualified commercially insured patients and a patient assistance program for uninsured or underinsured patients. Generic carglumic acid tablets are FDA approved and listed in the FDA's NDC Directory, labeled for NAGS deficiency only.
Help paying for Carbaglu
With private insurance, the drugmaker's copay card can lower what you pay for Carbaglu. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE, but charity funds often can. Of the 6 charity funds for the conditions it treats, 2 were open on October 26, 2026. Without insurance, you can apply to the drugmaker's free medicine program, which sets its own eligibility rules. Pick your insurance below to see which offers fit you.
- Copay help
Recordati offers a $0 copay for qualified patients with commercial insurance. No annual cap or Medicare and Medicaid exclusions are published on the page.
For: private insurance · source - Free medicine program
A Patient Assistance Program helps with access to medication for people who do not have insurance or are underinsured. Eligibility requirements may apply; no income or other terms are published.
For: no insurance, underinsured · source - Insurance and case manager help
Insurance verification, prior authorization support, alternate funding referrals, home delivery, and a one-on-one patient liaison for eligible NAGS deficiency patients and caregivers.
The official page does not say who qualifies. Ask the program. · source
Good to know: The U.S. number is 1-888-454-8860 and the Canada number is 1-844-322-7224; no hours are published. The page describes the patient liaison for NAGS deficiency patients and does not say whether every service also covers propionic or methylmalonic acidemia. Generic carglumic acid is FDA approved for NAGS deficiency.
Charity funds
- From a charity · The Assistance FundUrea Cycle Disorders (UCD) fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
- From a charity · NORD RareCareOrganic Acidemia Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · NORD RareCareOrganic Acidemia Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for current year”
Every program and charity fund for Urea Cycle Disorders →
Access and eligibility
Approved for adults and children, from birth, with acute or chronic high ammonia due to NAGS deficiency, and as an add-on to standard care for acute high ammonia due to propionic acidemia or methylmalonic acidemia[1]. For PA and MMA the label limits treatment to the acute episode, at most 7 days, and the label says treatment for NAGS deficiency starts as soon as the diagnosis is suspected[1]. Clinical studies did not include anyone 65 or older[1].
Source: Carbaglu Patient Support (Recordati Rare Diseases)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
For NAGS deficiency, approval rested on a retrospective case series of 23 patients treated for a median of 7.9 years, most of whom started as babies[1]. In the 13 patients with ammonia levels recorded before and after treatment, the mean ammonia level fell from 271µmol/L at baseline to 27µmol/L by day 3 in the 5 patients with a day 3 reading, and stayed at a mean of 23µmol/L in all 13 on long-term treatment, against a normal range of 5 to 50µmol/L[1].
The label notes this data was collected after the fact, unblinded and without a comparison group[1]. For PA and MMA, a randomized, double-blind, placebo-controlled trial (NCT01599286) treated 90 hospital episodes of high ammonia in 24 patients aged 4 days to 29 years, with every episode also getting standard care[1][4]. The median time to reach normal ammonia or hospital discharge was 1.5 days on Carbaglu against 2.0 days on placebo, a difference of 0.5 days whose 95% confidence interval ran from minus 1.2 to plus 0.1 days[1].
Development history
The FDA approved Carbaglu on March 18, 2010 under NDA 022562 as a new molecular entity, with priority review and orphan status, for high ammonia due to NAGS deficiency[2]. On January 22, 2021 the FDA approved new indications for acute high ammonia due to propionic acidemia or methylmalonic acidemia[2], and Recordati Rare Diseases said in its January 26, 2021 release that Carbaglu was the first and only FDA-approved medicine for that use[3].
The label updated in January 2024 reports dosing for patients with kidney impairment[1]. The FDA has approved generic carglumic acid tablets from Novitium Pharma (October 2021) and Navinta (June 2022), both rated as equivalent to Carbaglu[2][5]. The generic labels list only NAGS deficiency, not PA or MMA[6][7].
Explore Urea Cycle Disorders and Methylmalonic Acidemia trials
Common questions about Carglumic acid
▸What is Carglumic acid (Carbaglu)?
An oral tablet, mixed in water, that lowers dangerously high blood ammonia. Carbaglu was approved in 2010 for NAGS deficiency, a urea cycle disorder, both for sudden ammonia spikes and for long-term control. Since January 2021 it is also approved, alongside standard hospital care, for sudden high ammonia in propionic acidemia (PA) and methylmalonic acidemia (MMA). The Carbaglu label has no boxed warning.
▸How does Carglumic acid work?
The liver clears ammonia, a toxic waste product of protein, through a chain of reactions called the urea cycle, which turns ammonia into urea that leaves the body in urine. The first enzyme in that chain, carbamoyl phosphate synthetase 1 (CPS1), only works when a helper molecule called N-acetylglutamate (NAG) switches it on[1]. NAG is made by an enzyme called N-acetylglutamate synthase (NAGS), and people with NAGS deficiency cannot make enough of it, which leaves the urea cycle stalled and ammonia rising[1]. Carglumic acid is a man-made copy of NAG's shape that switches CPS1 on in its place, which the label says improves or restores urea cycle function and helps the body clear ammonia[1]. In propionic acidemia and methylmalonic acidemia, Carbaglu is used only during acute ammonia spikes, on top of standard hospital treatment[1].
▸What are the side effects of Carglumic acid?
The Carbaglu label has no boxed warning, no contraindications and no Warnings and Precautions section[1]. In the NAGS deficiency case series, 17 of 23 patients reported a side effect, and the most common were vomiting (26%), abdominal pain (17%), fever (17%), tonsillitis (17%) and, at 13% each, anemia, diarrhea, ear infection, infections, cold symptoms, low hemoglobin and headache[1]. In the PA and MMA trial, counted per hospital episode, at least 1 side effect was reported in 42% of episodes, and those more common with Carbaglu than placebo included neutropenia, a drop in infection-fighting white blood cells (14% vs 8%), anemia (12% vs 8%), vomiting (7% vs 2%), electrolyte imbalance (7% vs 4%), low blood sugar (5% vs 2%), decreased appetite (5% vs 2%), lethargy or stupor (5% vs 2%), encephalopathy, a brain function problem (5% vs 0), and pancreatitis or raised lipase (5% vs 0)[1]. Since approval, mania, itching and rashes have been reported, without a known rate[1]. One patient given 650mg/kg a day developed a fast heart rate, heavy sweating, extra airway mucus, raised temperature and restlessness, which resolved when the dose came down[1]. For pregnancy, the label says case reports are too few to judge risk, notes that untreated NAGS deficiency, PA and MMA can cause irreversible brain damage and death in pregnant women, and points to a pregnancy exposure registry; it is not known whether carglumic acid passes into human milk[1].
▸How is Carglumic acid taken?
Carbaglu comes as 200mg tablets for oral suspension, scored to split into 4 equal parts[1]. The tablets are stirred into water, at least 2.5mL per whole, half or quarter tablet, and taken right away, just before meals or feedings; they are not swallowed whole or crushed, and the label says mixing in foods or liquids other than water has not been studied and is not recommended[1]. The mixture can be swallowed from a cup, given by oral syringe, or given through a nasogastric or gastrostomy feeding tube[1]. For NAGS deficiency, the label dose is 100mg/kg to 250mg/kg a day for acute high ammonia and 10mg/kg to 100mg/kg a day for maintenance, split into 2 to 4 doses and adjusted to keep ammonia in the normal range for age[1]. For acute high ammonia in PA or MMA, the dose is 150mg/kg a day for patients 15kg or less, or 3.3g per square meter of body surface area a day for patients over 15kg, split into 2 doses 12 hours apart, until ammonia falls below 50µmol/L and for no more than 7 days[1]. Doses are lower for moderate or severe kidney impairment[1]. Unopened bottles go in the refrigerator; once opened, the bottle is kept at room temperature, tightly closed, and thrown away 1 month after opening[1].
▸Is Carglumic acid FDA approved?
Yes, Carglumic acid (Carbaglu) is FDA approved (2010) for the treatment of Urea Cycle Disorders and Methylmalonic Acidemia.
▸What is Carbaglu used for?
Carbaglu (carglumic acid) lowers high blood ammonia in NAGS deficiency, a urea cycle disorder, and in sudden ammonia spikes from propionic acidemia or methylmalonic acidemia.
NAGS deficiency: Approved for sudden high ammonia, alongside standard care, and for long-term maintenance.
PA and MMA: Approved only as an add-on to standard care during acute high ammonia, for up to 7 days.
Ages: Adults and children, from birth.
Source: Carbaglu prescribing information
▸How do you take Carbaglu tablets?
Carbaglu 200mg tablets are stirred into water and taken right away, just before meals or feedings.
Water: At least 2.5mL for each whole, half or quarter tablet; the tablets do not fully dissolve, and the label says to rinse the cup with more water and swallow that too.
Not allowed: Swallowing tablets whole, crushing them, or mixing them in other foods or drinks.
Feeding tubes: The mixture can go through a nasogastric or gastrostomy tube, flushed with 1 to 2mL of water.
Storage: Refrigerate until opened, then keep at room temperature and discard 1 month after opening.
Source: Carbaglu prescribing information
▸Is there a generic Carbaglu?
Yes. The FDA has approved generic carglumic acid tablets rated as equivalent to Carbaglu.
Makers: Novitium Pharma (approved October 2021) and Navinta (approved June 2022), with labels also listed under Eton Pharmaceuticals and Burel Pharmaceuticals.
Uses: The generic labels list NAGS deficiency only, while Carbaglu's label also covers acute high ammonia in propionic and methylmalonic acidemia.
Source: Drugs@FDA, NDA 022562 and generic carglumic acid label
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed (Recordati Rare Diseases) · 2024-01. CARBAGLU- carglumic acid tablet, for suspension. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=10175e73-5172-4dde-a508-8a88b7afc0a1
- U.S. Food and Drug Administration · 2010-03-18. CARBAGLU approval history, NDA 022562. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=022562
- Recordati Rare Diseases (PR Newswire, via BioSpace) · 2021-01-26. RECORDATI RARE DISEASES: CARBAGLU® (Carglumic Acid) Tablets 200mg Receives U.S. FDA Approval for a New Indication to Treat Acute Hyperammonemia Associated with Propionic Acidemia and Methylmalonic Acidemia. https://www.biospace.com/recordati-rare-diseases-carbaglu-carglumic-acid-tablets-200mg-receives-u-s-fda-approval-for-a-new-indication-to-treat-acute-hyperammonemia-associated-with-propionic-acidemia-and-methylmalonic-acidemia
- ClinicalTrials.gov. Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia. https://clinicaltrials.gov/study/NCT01599286
- U.S. Food and Drug Administration, openFDA · 2026-11-12. Drugs@FDA records for carglumic acid (NDA 022562, ANDA 213729, ANDA 213395). https://api.fda.gov/drug/drugsfda.json?search=products.active_ingredients.name:%22CARGLUMIC+ACID%22&limit=50
- U.S. National Library of Medicine, DailyMed (Eton Pharmaceuticals) · 2026-03. Carglumic acid tablets for oral suspension: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=591f95ca-3d99-4fb6-a402-e5a383a746d0
- U.S. National Library of Medicine, DailyMed (Horizon Therapeutics USA) · 2025-10. RAVICTI (glycerol phenylbutyrate) oral liquid: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=900e7dc0-9afe-4f50-80de-90567bb78519
- U.S. National Library of Medicine, DailyMed (Horizon Therapeutics USA) · 2026-11. BUPHENYL (sodium phenylbutyrate): prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=463a36fa-3eb2-4326-8bd0-c8c7a11bca3a
- Recordati Rare Diseases. CARBAGLU® (carglumic acid) │ Patient Support. https://www.carbaglu.com/dtc/nags-deficiency-support/