Pharmacy Prior Authorization Criteria

Procysbi (cysteamine bitartrate)

For diagnosis of Nephropathic cystinosis
Indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

AND

AND

Trial and failure or intolerance to Cystagon (immediate-release cysteamine bitartrate) [B, 4-10]

AND

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

Patient demonstrates positive clinical response to therapy (e.g., decrease in cystine levels in white blood cells)

2025-08-12, 2024-06-05, 2023-10-12, 2023-06-06, 2022-06-02, 2021-06-02, 2020-05-05, 2020-03-24

Procysbi Prescribing Information. Horizon Pharma USA, Inc. Lake Forest, IL. February 2022.
Emma F, Nesterova G, Langman C, et al. Nephropathic cystinosis: an international consensus document. Nephrol Dial Transplant. 2014 Sep;29(Suppl 4): iv87–iv94.
Wilmer MJ, Schoeber JP, van den Heuvel LP, Levtchenko EN. Cystinosis: practical tools for diagnosis and treatment [educational review]. Pediatr Nephrol 2011 Feb; 26(2): 205-15.
Medic G, van der Weijden M, Karabis A, Hemels M. A systematic literature review of cysteamine bitartrate in the treatment of nephropathic cystinosis. Curr Med Res Opin. 2017; 33(11):2065-2076.
Niebl C, Boulesteix AL, Oh J, et al. Relationship between age at initiation of cysteamine treatment, adherence with therapy, and glomerular kidney function in infantile nephropathic cystinosis. Mol Genet Metab. 2022; 136(4): 268-273.
Chang HE, Hossain MS, Song C, et al. Long-term outcomes in nephropathic cystinosis: a review. Pediatr Nephrol. 2025 May 14. doi: 10.1007/s00467-025-06790-6. Online ahead of print.
Per clinical consult with a nephrologist, July 10, 2025.
Per clinical consult with a nephrologist, July 18, 2025.
van Stein C, Klank S, Gruneberg M, et al. A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis. Orphanet J Rare Dis. 2021; 16(1):387.
Ariceta G, Lalanza S, Pena, C, et al. Patient journey in cystinosis: focus on non-adherence and disease management. Drugs Context. 2024 Nov 18: 13:2024-7-1.

A definitive diagnosis can be verified by measuring leukocyte cystine levels, genetic analysis of the CTNS gene or demonstration of corneal crystals by slit lamp examination. [2, 3]
Cystagon is an immediate release cysteamine bitartrate product indicated for nephropathic cystinosis in children of all ages and adults. Symptoms of nephropathic cystinosis arise in the first 3 to 12 months of life, and patients are usually diagnosed within the first 2 years of life. [4-6] Cysteamine therapy should be initiated as early as possible upon diagnosis due to the severity of disease, including kidney failure by 9 years of age and failure across various other organs without treatment. While there is no statistically significant difference in effectiveness of the immediate release (i.e., Cystagon) and delayed release (i.e., Procysbi) cysteamine formulations when patients are adherent to therapy, the dosing regimen of Cystagon is often a barrier in real-world settings. [7-9] Non-adherence is most often observed during adolescence, as caregivers are mainly in charge of and maintain adherence with treatment throughout patients’ childhood, although non-adherence is also observed in adulthood. [7, 9, 10]

2025-08-12: Annual Review 2025 - Program update for standard UM verbiage with no change to clinical intent.
2024-06-05: Annual Review - No criteria changes
2023-10-12: Program update to standard reauthorization language. No changes to clinical intent.
2023-06-06: Annual Review - no criteria changes
2022-06-02: Annual review - Addition of reauth criteria. Approval durations modified.
2021-06-02: 2021 Annual Review, no changes to criteria.
2020-05-05: Annual review, no changes to clinical criteria.
2020-03-24: Addition of granule products