NDAORALCAPSULE, DELAYED RELEASE
Approved
Apr 2013
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. First approved in 2013.
Drug data last refreshed 4h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
PROCYSBI (cysteamine bitartrate) is an oral delayed-release capsule that treats nephropathic cystinosis, a rare autosomal recessive lysosomal storage disorder. The drug works by participating in a thiol-disulfide interchange within lysosomes to convert cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome. Without treatment, cystinosis causes progressive multi-organ damage including renal failure by age 10, growth failure, rickets, photophobia, and eventual involvement of the retina, muscles, and central nervous system.
$39MPart D
PeakStable
100% of nephropathic cystinosis treatment market (no direct competitors)
10.7 years to LOEOrphan drug at peak maturity with concentrated patient base and stable revenue; small specialized team focused on rare disease management.
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 (6)
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.