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Cysteamine Bitartrate Delayed-release Capsules
Cysteamine bitartrate reduces cystine accumulation in lysosomes by converting cystine to cysteine and cysteamine, thereby decreasing intracellular cystine levels.
Cysteamine bitartrate reduces cystine accumulation in lysosomes by converting cystine to cysteine and cysteamine, thereby decreasing intracellular cystine levels. Used for Cystinosis (nephropathic cystinosis).
At a glance
| Generic name | Cysteamine Bitartrate Delayed-release Capsules |
|---|---|
| Also known as | RP103, PROCYSBI® |
| Sponsor | Amgen |
| Drug class | Cystine-depleting agent |
| Target | Lysosomal cystine |
| Modality | Small molecule |
| Therapeutic area | Rare Genetic Disorder |
| Phase | Phase 3 |
Mechanism of action
Cystinosis is a lysosomal storage disorder caused by mutations in the CTNS gene, leading to cystine accumulation in lysosomes and cellular damage. Cysteamine acts as a reducing agent that converts the disulfide cystine into two molecules of cysteine and cysteamine, which can then exit the lysosome via alternative transporters. This mechanism reduces toxic cystine levels and slows progression of organ damage, particularly in the kidneys.
Approved indications
- Cystinosis (nephropathic cystinosis)
Common side effects
- Gastrointestinal disturbances (nausea, vomiting, diarrhea)
- Abdominal pain
- Headache
- Rash
- Fever
Key clinical trials
- Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis (PHASE3)
- A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease (PHASE2)
- Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis (PHASE3)
- Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) (PHASE3)
- Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease (PHASE2)
- Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis (PHASE1, PHASE2)
- Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis (PHASE3)
- Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children (PHASE2, PHASE3)
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 |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |