Last reviewed · How we verify
Cystagon (Mercaptamine)
Cystagon works by binding to cystine and forming a disulfide bond, effectively removing cystine from the body.
At a glance
| Generic name | Mercaptamine |
|---|---|
| Sponsor | Mylan |
| Drug class | Cystine Depleting Agent |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 1994 |
Mechanism of action
Mechanism of Action. Cystinosis is an autosomal recessive inborn error of metabolism in which the transport of cystine out of lysosomes is abnormal; in the nephropathic form, accumulation of cystine and formation of crystals damage various organs, especially the kidney, leading to renal tubular Fanconi Syndrome and progressive glomerular failure, with end stage renal failure by the end of the first decade of life. In four studies of cystinosis patients before cysteamine was available, renal death (need for transplant or dialysis) occurred at median age of less than 10 years. Patients with cystinosis also experience growth failure, rickets, and photophobia due to cystine deposits in the cornea. With time most organs are damaged, including the retina, muscles and central nervous system.Cysteamine is an aminothiol that participates within lysosomes in thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the l
Approved indications
- Cystinosis
Common side effects
- vomiting
- anorexia
- fever
- diarrhea
- lethargy
- rash
Key clinical trials
- Effectiveness of a Bilayering Serum and Cream Containing GABA, DMAE, Cysteamine, and Bakuchiol for Skin Whitening and Anti-Aging (PHASE2)
- Use of Cysteamine in the Treatment of Cystinosis
- Cystinosis and Mitochondrial Metabolism (NA)
- A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications (NA)
- PK and PD Study of NPI-001 and Cysteamine Bitartrate (PHASE1,PHASE2)
- DFT383 in Pediatric Participants With Nephropathic Cystinosis (PHASE1,PHASE2)
- A Study to Assess TTI-0102 vs Placebo in MELAS Patients (PHASE2)
- A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS) (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Cystagon CI brief — competitive landscape report
- Cystagon updates RSS · CI watch RSS
- Mylan portfolio CI