NDAORALGRANULE, DELAYED RELEASE
Approved
Feb 2020
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
6
Data completeness
5/8 — Partial
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 2020.
Drug data last refreshed 19h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
PROCYSBI (cysteamine bitartrate) is a delayed-release oral granule approved in 2020 for cystinosis, a rare autosomal recessive lysosomal storage disorder. It works by participating in thiol-disulfide interchange reactions within lysosomes to convert toxic cystine into cysteine and mixed disulfides that can exit the lysosome. This mechanism halts progression of multi-organ damage caused by cystine crystal accumulation.
Peak10.8 years to LOE
PROCYSBI is in peak commercial phase with $39M spending, indicating active market presence and sustained team investment for this orphan indication.
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 (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.