Manages nephropathic cystinosis. Sold as Cystagon, Procysbi. Every FDA application for this active ingredient.
As of Nov 15, 2026, FDA lists 3 applications for Cysteamine Bitartrate (sold as Cystagon, Procysbi), first approved Aug 15, 1994, with 2 new uses approved and 0 published complete response letters.
| Procysbi | Part D | $40.9M | +8% | 55 |
| Cystagon | Part D | $57K | -3% | 15 |
| Pilot Study of TTI-0102 in CystinosisNephropathic Cystinosis, Cystinosis · Thiogenesis Therapeutics, Inc. | Phase 2 | Not yet recruiting | Sep 23, 2026 |
| European Cystinosis Cohort 2Cystinosis · Institut National de la Santé Et de la Recherche Médicale, France | — | Not yet recruiting | Jul 2, 2026 |
| Cystinosis and Mitochondrial MetabolismCystinosis, Native Kidney · Hospices Civils de Lyon | Not applicable | Recruiting | Jan 6, 2026 |
| A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)Leigh Syndrome · Thiogenesis Therapeutics, Inc. | Phase 2 | Not yet recruiting | May 25, 2025 |
| DFT383 in Pediatric Participants With Nephropathic CystinosisNephropathic Cystinosis · Novartis Pharmaceuticals | Phase 1 / Phase 2 | Recruiting | Apr 4, 2025 |
| Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community- Acquired PneumoniaRespiratory Tract Diseases, Influenza (Pandemic) · UMC Utrecht | Phase 3 | Recruiting | Apr 13, 2016 |
| Use of Cysteamine in the Treatment of CystinosisCystinosis · National Human Genome Research Institute (NHGRI) | — | Recruiting | Aug 2, 2006 |
Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA
Mylan