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 |
|---|---|---|---|---|---|
2021/09/10 | N/A | Recruiting | |||
2019/11/25 | N/A | Recruiting | |||
2015/04/27 | Phase 3 | Completed | |||
2012/05/16 | Phase 2 | Completed | Mendel Tuchman | ||
2012/05/14 | Phase 2 | Terminated | Mendel Tuchman | ||
2011/04/25 | Phase 2 | Withdrawn | |||
2009/02/13 | Phase 2 | UNKNOWN | Mendel Tuchman |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Navinta LLC | 68475-006 | ORAL | 200 mg in 1 1 | 2/10/2023 | |
| Eton Pharmaceuticals, Inc. | 71863-114 | ORAL | 200 mg in 1 1 | 1/31/2024 | |
| Burel Pharmaceuticals, LLC | 35573-459 | ORAL | 200 mg in 1 1 | 3/19/2024 | |
| Recordati Rare Diseases | 52276-312 | ORAL | 200 mg in 1 1 | 1/25/2024 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 6/23/2017 | ||
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 |
|---|---|---|---|---|---|
| Carglumic Acid Dispersible Tablets | 国药准字H20234128 | 化学药品 | 片剂 | 9/5/2023 | |
| Carglumic Acid Dispersible Tablets | 国药准字HJ20230085 | 化学药品 | 片剂 | 6/27/2023 |
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 | Medicine | A | 2/12/2015 |
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