CurrentUnitedHealthcarePolicy N/A

Step therapy for Procysbi (cysteamine bitartrate)

UnitedHealthcare step therapy program requiring trial of Cystagon (cysteamine bitartrate) prior to coverage of Procysbi for management of nephropathic cystinosis in patients aged 1 year and older; continuation allowed for members already on Procysbi per claims history.

Policy Summary

PayerUnitedHealthcare

PolicyStep therapy for Procysbi (cysteamine bitartrate)

Policy CodePolicy N/A

Change TypeClarifications and administrative updates

Effective DateSep 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization for Procysbi and document either failure/contraindication/intolerance to Cystagon or evidence of current Procysbi use in claims history; users established only by manufacturer samples or assistance must meet initial authorization criteria.

SourceLink

POLICY UPDATE CHANGES

Duration of authorization was updated to 12 months (9/2022).

Updated name of manufacturer assistance program referenced in sample/assistance language (9/2023, 9/2024, 6/2025).

Procysbi approval age clarified historically (background noted approval for pediatric patients 1 year and older in current text).

12Authorization duration

1Minimum patient age for indication

2Approval clinical paths

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Medically Necessary / Procysbi approval criteria

Procysbi will be approved when ONE of the following is met:

ANY of the following

History of failure, contraindication, or intolerance to Cystagon (cysteamine bitartrate).
Current user exception
- Patient is currently on Procysbi therapy (documented in claims history).
- Patient has not received a manufacturer supplied sample at no cost in the prescriber's office, or any form of assistance from the Amgen By Your Side sponsored program (e.g., sample card redeemable at a pharmacy) as a means to establish current user status.
  If patient was 'established' on therapy solely via receipt of a manufacturer sample or assistance program, they must meet initial authorization criteria as if new to therapy.

What Providers Must Do

Prior Authorization

Step therapy prior authorization required

Providers must obtain prior authorization for Procysbi for members new to therapy and demonstrate either failure/contraindication/intolerance to Cystagon or that the member is a documented current Procysbi user (with restrictions around manufacturer samples/assistance).

Documentation Required

Document prior failure or current therapy

Providers should document history of failure, contraindication, or intolerance to Cystagon, or provide evidence in claims history that the patient is a current Procysbi user; if current use is based solely on manufacturer samples or assistance program benefits, initial authorization criteria must be met.

Denial Risk

Denial risk for sample-established users

Requests for initial authorization where the only evidence of prior Procysbi use is receipt of manufacturer samples or assistance program benefits will be processed as new therapy and may be denied if initial criteria are not met.

Clinical and Program Background

Step therapy is used to encourage use of lower-cost alternatives. This program requires a trial of Cystagon (cysteamine bitartrate) before coverage is provided for Procysbi (cysteamine bitartrate) for the management of nephropathic cystinosis. Both Procysbi and Cystagon are indicated for adults and pediatric patients 1 year of age and older.

Members who are currently on Procysbi therapy as documented in claims history will be allowed to continue their current therapy. However, if a member’s only evidence of prior Procysbi use is receipt of a manufacturer-supplied sample or assistance (for example, via the Amgen By Your Side program or a sample card redeemed at a pharmacy) used to establish current user status, that member must meet initial authorization criteria as if new to therapy.

Operationally, approved authorizations will be issued for 12 months. UnitedHealthcare may apply automated approval or reauthorization based on claims history, diagnosis codes (ICD-10) and claim logic; state mandates, federal requirements, member-specific benefit plans, and other utilization management programs may also affect coverage. Notifications and supply limits may apply.

Authorization and Operational Details

Authorization length12 months.

State and plan considerationsState mandates, federal regulatory requirements, and member-specific benefit plans may affect coverage.

Automated approvals and re-approvalUnitedHealthcare may use automated approval/re-approval based on claims history, diagnosis codes (ICD-10), and claim logic; use varies by program.

Notifications and supply limitsNotifications and supply limits may apply as part of utilization management.

Authorization duration12 months

Minimum patient age for indication1 year

Ways to meet approval2 clinical paths: (1) history of failure/contraindication/intolerance to Cystagon OR (2) current Procysbi user documented in claims history (with restrictions for sample-established users).

Policy Updates

2022-09authorization_duration_update

Duration of authorization was updated to 12 months.

2023-09assistance_program_name_update

Updated name of manufacturer assistance program referenced in sample/assistance language; no change to coverage criteria.

2024-09annual_review

Annual review with no changes to coverage criteria; background and references updated.

2025-06assistance_program_name_updateLatest

Updated name of manufacturer assistance program referenced in sample/assistance language.

Historical (prior to current text)background_clarification

Background clarified that Procysbi is indicated for pediatric patients 1 year of age and older.