NDAORALCAPSULE, DELAYED RELEASE
Approved
Apr 2013
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
6
Data completeness
6/8 — Strong
PROCYSBI (cysteamine bitartrate) by Amgen 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. Approved for cystinosis. First approved in 2013.
Drug data last refreshed 20h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
PROCYSBI (cysteamine bitartrate) is an oral, delayed-release capsule approved by the FDA in 2013 for the treatment of nephropathic cystinosis in children. The drug works by participating in a thiol-disulfide interchange reaction within lysosomes, converting cystine into cysteine and cysteine-cysteamine mixed disulfide, which can then exit the lysosome. Cystinosis is a rare autosomal recessive lysosomal storage disorder in which cystine accumulation causes progressive kidney damage, growth failure, and multi-organ pathology. PROCYSBI is the formulation of choice in the cystinosis market, offering improved dosing convenience over older immediate-release cysteamine formulations.
$0.0BPart D
10.8 years to LOEMechanism 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 (1)
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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.