carglumic acid
Trade name: carbaglu
Approved
Mar 18, 2010
Carglumic acid is a Carbamoyl Phosphate Synthetase 1 (CPS 1) allosteric modulator. CPS1 is found in the mitochondria and is the first enzyme of the urea cycle, which converts ammonia into urea. Carglumic acid acts as a replacement for NAG in NAGS deficiency patients by activating CPS1 but it does not help to regulate the urea cycle. Carglumic acid under the trade name Carbaglu indicated as adjunctive therapy for the treatment of acute hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS). In addition, as maintenance therapy for the treatment of chronic hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS). 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. — NCATS
Clinical trial activity
6 trials · 14 clinical orgs · 5 marketing orgs
Earliest trial started Aug 1, 2008 (NCT00843921)
Timeline
1990s
- Jan 20, 1998
Orphan Drug Designation
2000s
- Jan 1, 2008
Earliest Phase 2 Sponsor(trial)
2010s
- Mar 18, 2010
Earliest FDA Approval
- Mar 18, 2010
Orphan Europe — NDA Secondary Org
- Mar 18, 2010
Recordati — NDA Secondary Org
- Jan 1, 2015
Earliest Phase 3 Sponsor(trial)
2020s
- Oct 13, 2021
Novitium Pharma — Marketing Organization
- Oct 13, 2021
Ani Pharmaceuticals — Marketing Organization
- Jun 22, 2022
Navinta — Marketing Organization
Indications
Approved for
Mechanism of action
- Carbamoyl-phosphate synthase [ammonia], mitochondrialPOSITIVE ALLOSTERIC MODULATOR
CPS1 positive allosteric modulator
Approval history
- approvedPriority reviewMar 18, 2010
Chemistry & pharmacology
SMILES
NC(=O)N[C@@H](CCC(O)=O)C(O)=O- Mol. weight
- 190.154 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
Sources
- WikipediaCarglumic acid ↗
- NCATS5L0HB4V1EW ↗
- ChEMBLCHEMBL1201780 ↗
Also known as
- (2s)-2-(carbamoylamino) pentanedioic acid
- (2s)-2-(carbamoylamino)pentanedioic acid
- (2s)-2-(carbamoylamino)pentanedioic acid
- acide carglumique
- acide carglumique
- acido carglumico
- acido carglumico
- acidum carglumicum
- acidum carglumicum
- carbaglu
- carbaglu
- carbaglu
- carbaglu
- carbaglu
- carbaglu
- carbamino-l-glutamic acid
- carbamino-l-glutamic acid
- carbamylglutamic acid
- carbamylglutamic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- carglumic acid
- l-n-carbamoylglutamic acid
- l-n-carbamoylglutamic acid
- n-carbamoyl-l-glutamic acid
- n-carbamoyl-l-glutamic acid
- n-carbamylglutamate
- n-carbamylglutamate
- n-carbamylglutamate
- n-carbamyl-l-glutamate
- n-carbamyl-l-glutamate
- oe-312 (laboratory-code-designation)
- oe-312 (laboratory-code-designation)
- (s)-2-ureidopentanedioic acid
- (s)-2-ureidopentanedioic acid
- ureidoglutaric acid
- ureidoglutaric acid
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04284917 | N/A | Nov 28, 2019 | National Taiwan University |
| NCT02426775 | Phase 3 | Nov 1, 2015 | King Abdul-Aziz University |
| NCT01597440 | Phase 2 | Sep 1, 2012 | Case Western Reserve University, Children's National Medical Center, Children's National Research Institute, Harvard University, Stanford University, University of California, Los Angeles, University of Colorado, Denver, University of Pennsylvania |
| NCT01599286 | Phase 2 | Sep 1, 2012 | Case Western Reserve University, Children's National Medical Center, Children's National Research Institute, Harvard University, Icahn School of Medicine at Mount Sinai, Stanford University, University of California, Los Angeles, University of Colorado, University of Pennsylvania, University of Pittsburgh |
| NCT01341379 | Phase 2 | Dec 1, 2010 | National Institutes of Health (NIH), University of Pennsylvania |
| NCT00843921 | Phase 2/Phase 3 (Phase 3) | Aug 1, 2008 | Children's National Medical Center, Children's National Research Institute, National Institutes of Health (NIH) |
Organizations
Research & Development (14)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Children's National Medical Center | Academic/Hospital | 3 | 0 | 2 | 2008 |
| Children's National Research Institute | Academic/Hospital | 3 | 3 | 2 | 2008 |
| University of Pennsylvania | Academic/Hospital | 3 | 1 | 1 | 2010 |
| Case Western Reserve University | Academic/Hospital | 2 | 0 | 1 | 2012 |
| Harvard University | Academic/Hospital | 2 | 0 | 1 | 2012 |
| National Institutes of Health (NIH) | Government | 2 | 0 | 2 | 2008 |
| Stanford University | Academic/Hospital | 2 | 0 | 1 | 2012 |
| University of California, Los Angeles | Academic/Hospital | 2 | 0 | 1 | 2012 |
| Icahn School of Medicine at Mount Sinai | Academic/Hospital | 1 | 0 | 1 | 2012 |
| King Abdul-Aziz University | Academic/Hospital | 1 | 1 | 1 | 2015 |
| National Taiwan University | Academic/Hospital | 1 | 1 | 1 | 2019 |
| University of Colorado | Academic/Hospital | 1 | 0 | 1 | 2012 |
| University of Colorado, Denver | Academic/Hospital | 1 | 0 | 1 | 2012 |
| University of Pittsburgh | Academic/Hospital | 1 | 0 | 1 | 2012 |
Marketing (6)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Ani Pharmaceuticals | For profit | MKTG | Oct 13, 2021 |
| Navinta | For profit | MKTG | Jun 22, 2022 |
| Novitium Pharma | For profit | MKTG | Oct 13, 2021 |
| Orphan Europe | For profit | NDA2 | Mar 18, 2010 |
| Recordati | For profit | NDA | — |
| Recordati | For profit | NDA2 | Mar 18, 2010 |