Carglumic acid

Overview

Carglumic acid is a drug used for the treatment of hyperammonemia in patients with a deficiency in N-acetyl glutamate synthase. This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood–brain barrier and cause neurologic problems, cerebral edema, coma, and death. Carglumic acid was approved by the U.S. Food and Drug Administration (FDA) on 18 March 2010.

Indication

For the treatment of acute and chronic hyperammonaemia in patients with N-acetylglutamate synthase (NAGS) deficiency. This enzyme is an important component of the urea cycle to prevent build up of neurotoxic ammonium in the blood.

Associated Conditions

Acute Hyperammonaemia
Chronic Hyperammonaemia

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics	2021/09/10	NCT05040178	N/A	Recruiting	Recordati Rare Diseases
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach	2019/11/25	NCT04176523	N/A	Recruiting	Recordati Rare Diseases
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia	2015/04/27	NCT02426775	Phase 3	Completed	King Abdullah International Medical Research Center
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia	2012/05/16	NCT01599286	Phase 2	Completed	Mendel Tuchman
Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	2012/05/14	NCT01597440	Phase 2	Terminated	Mendel Tuchman
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate	2011/04/25	NCT01341379	Phase 2	Withdrawn	Children's Hospital of Philadelphia
N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia	2009/02/13	NCT00843921	Phase 2	UNKNOWN	Mendel Tuchman

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Carglumic acid	Navinta LLC	68475-006	ORAL	200 mg in 1 1	2/10/2023
Carglumic Acid	Eton Pharmaceuticals, Inc.	71863-114	ORAL	200 mg in 1 1	1/31/2024
Carglumic acid	Burel Pharmaceuticals, LLC	35573-459	ORAL	200 mg in 1 1	3/19/2024
Carbaglu	Recordati Rare Diseases	52276-312	ORAL	200 mg in 1 1	1/25/2024

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Ucedane	Eurocept International BV,Trapgans 5,1244 RL Ankeveen,The Netherlands	Authorised	6/23/2017
Carbaglu	Tour Hekla,Tour Hekla,52 avenue du Général de Gaulle,F-92800 Puteaux,France	Authorised	1/24/2003

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
Carbaglu carglumic acid 200 mg dispersible tablets tube	215632	Recordati Rare Diseases Australia Pty Ltd	Medicine	A	2/12/2015

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
CARBAGLU	RECORDATI RARE DISEASES	02439360	Tablet For Suspension - Oral	200 MG	9/8/2015

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
No CIMA AEMPS (Spain) approvals found for this drug.

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

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