Carglumic Acid

Treats acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Sold as Carbaglu. Every FDA application for this active ingredient: 1 branded and 2 generic.

As of Nov 15, 2026, FDA lists 3 applications for Carglumic Acid (sold as Carbaglu), first approved Mar 18, 2010, with 2 new uses approved and 0 published complete response letters.

First approved: Mar 18, 2010
Applications: 3
New uses approved: 2
Complete response letters: 0
Medicare spending, 2025: $14.7M
Orphan exclusivity until: Jan 22, 2028
First approved in the EU: Jan 24, 2003
First approved in Japan: Sep 28, 2016

Medicare Spending

CMS Part D and Part B, 2025, before manufacturer rebates; matched by generic name


Carbaglu	Part D	$8.2M	-24%	0
Carglumic Acid	Part D	$6.5M	+1039%	68

Orphan Designations (FDA)

2 designations, 2 approved for the designated use; see all orphan designations

Treatment of organic acidemiasRecordati Rare Diseases · designated Jun 17, 2014 · approved Jan 22, 2021 · exclusive to Jan 22, 2028
Treatment of N-acetylglutamate synthetase deficiency.Orphan Europe SARL · designated Jan 20, 1998 · approved Mar 18, 2010

Approvals in the EU (EMA)

First approved in the EU 86 months before the US. Centrally authorised medicines, matched by ingredient; see all EU approvals

Ucedane, genericEurocept International BV · Jun 23, 2017
Carbaglu, new substanceRecordati Rare Diseases · Jan 24, 2003

Approvals in Japan (PMDA)

First approved in Japan 78 months after the US. PMDA's English list since 2008, matched by ingredient; see all Japanese approvals

Carbaglu Dispersible, new ingredientA drug with a new active ingredient indicated for the treatment of hyperammonemia due to N- acetylglutamate synthetase deficiency, isovaleric acidemia,… · Pola Pharma Inc. · Sep 28, 2016

Clinical Trials

3 open or ongoing studies name this ingredient as an intervention, including as a comparator; newest first. See all


An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and PediatricsHyperammonemia, Methylmalonic Acidemia · RECORDATI GROUP	—	Recruiting	Sep 10, 2021
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachMethylmalonic Acidemia, Propionic Acidemia · RECORDATI GROUP	—	Recruiting	Nov 25, 2019
Orphan Europe Carbaglu® Surveillance ProtocolN-acetylglutamate Synthase (NAGS) Deficiency · Nicholas Ah Mew	—	Recruiting	Jan 24, 2018

Applications

NDAs, BLAs and generic ANDAs for this ingredient, oldest first; each opens its full history


Mar 18, 2010	CarbagluNDA 22562 · New molecular entity	Recordati Rare	11	Marketed
Oct 13, 2021	Carglumic AcidANDA 213729	Novitium Pharma	2	Marketed
Jun 22, 2022	Carglumic AcidANDA 213395	Navinta LLC	1	Marketed

Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA