CPS1 Inhibitors
2 drugsAbout CPS1
Carbamoyl phosphate synthetase 1 (CPS1) is a mitochondrial enzyme in liver cells that catalyzes the first step of the urea cycle. This cycle is essential for removing toxic ammonia from the body. Dysfunctional CPS1 can lead to hyperammonemia and neurological damage.
CPS1 is a significant drug target due to its role in ammonia detoxification. There is currently no genetic evidence directly linking CPS1 to specific diseases. However, its essential function makes it a target for metabolic disorders.
CPS1 is targeted by two FDA-approved small molecule drugs: CARBAGLU (RECORDATI RARE) and CARGLUMIC ACID (NOVITIUM PHARMA). These drugs are used in metabolic disorders involving ammonia detoxification, highlighting CPS1's druggability.
Top CPS1 Drugs
The competitive landscape consists of two companies, RECORDATI RARE and NOVITIUM PHARMA, with approved drugs.
The limited number of players suggests relatively low barriers to entry, but also a small market.
CPS1 Drug Modality Landscape
Modalities
Routes of Administration
CPS1 is amenable to small molecule drugs, with oral options available for convenient dosing.
Exploring alternative modalities like biologics could offer differentiation in a small molecule-dominated space.
CPS1 Clinical Trials 2 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on CPS1. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 2 | 1 | 1 | 0 | 0 | 100% |
| Phase 3 | 1 | 1 | 0 | 0 | 100% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
CPS1 Drug Approval Timeline (2010 - 2021)
The first drug targeting CPS1 was approved in 2010, and the most recent in 2021, spanning 12 years.
The recent approval suggests continued interest, but the slow pace indicates a potentially saturated market.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 2 companies competing
- • Market share by company
Full Drug Portfolio
- • All 2 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 2 (0 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on CPS1. A trial counts if an approved drug acting on CPS1 is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.