Carglumic acid
As of the edition of 20 October 2026, Carglumic acid is a small molecule: marketed, first approved in 2003 (EU); acts on CPS1; US protection ends in 2028.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2019
- Kind
- Small molecule
- Brand name
- Carbaglu
- First approved
- 24 January 2003 (EU)
- Approvals on record
- 5 in 5 regions
- Holder of the most recent approval
- RECORDATI RARE DISEASES COMÉRCIO DE MEDICAMENTOS LTDA - ME; 4 holders in all
- Registered trials
- 8, 2 stopped early
- Disease areas
- Inherited and neonatal; Nutrition and metabolism; Nervous system
US protection
US protection ends in 2028.
The last listed US exclusivity ends in 2028. The application that gives this date is held by Recordati Rare Diseases.
This is the US only, and it is not the date generics or biosimilars can enter.
The exact date and the listings behind it
In the product1 listing counts towards this date. The exact date and each listing are in the product.
See the exact date and the 1 listing behind itRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| CPS1 | Carbamoyl-phosphate synthase [ammonia], mitochondrial | P31327 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 3 | 1 | 2 | 0 |
| Phase 2 | 3 | 1 | 0 | 2 |
| Phase 3 | 2 | 1 | 0 | 0 |
| All trials | 8 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Tour Hekla | Applicant | 2003 to now |
| Recordati Rare Diseases | Acquired holder | 2019 to now |