NDAORALCAPSULEPriority Review
Approved
Aug 1994
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
8
Data completeness
5/8 — Partial
Priority Review
CYSTAGON (cysteamine bitartrate) by Viatris is 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. First approved in 1994.
Drug data last refreshed 10h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
CYSTAGON (cysteamine bitartrate) is an oral aminothiol indicated for nephropathic cystinosis, a rare autosomal recessive lysosomal storage disorder. It works by converting cystine into cysteine and cysteine-cysteamine mixed disulfide within lysosomes, allowing these compounds to exit and reducing organ damage. Without treatment, cystinosis leads to end-stage renal failure by age 10, but cysteamine delays progression and reduces secondary complications including growth failure, rickets, and photophobia.
$0.075MPart D
LOE ApproachingSmall patient base with declining revenue trajectory suggests a mature, specialty-focused team managing a long-established rare disease product nearing loss of exclusivity.
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…
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (8)
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Started Oct 2023
12 enrolled
Cystinosis
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
Started May 2015
22 enrolled
Mitochondrial Diseases
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
Started Jan 2013
41 enrolled
Cystinosis
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
Started Dec 2012
17 enrolled
Cystinosis
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Started Aug 2010
60 enrolled
Cystinosis
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.