CurrentAvMedPolicy N/A

Procysbi (cysteamine bitartrate) prior authorization

Defines pharmacy prior authorization and step-edit requirements for Procysbi (cysteamine bitartrate) delayed-release for members with nephropathic cystinosis, including initial authorization and reauthorization clinical criteria and required documentation.

Policy Summary

PayerAvMed

PolicyProcysbi (cysteamine bitartrate) prior authorization

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionSubmit documentation supporting each checked clinical criterion, including WBC cystine and serum creatinine labs, genetic test results or leukocyte cystine concentration, chart notes documenting intolerance to prior Cystagon therapy, and current height and weight.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsInitial authorization duration

12 monthsReauthorization duration

Age ≥1 yrMinimum member age

1.95 g/m2/dayMax dose

>2 nmolBaseline WBC cystine

Policy Summary

PayerAvMed

PolicyProcysbi (cysteamine bitartrate) prior authorization

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

SourceLink

On This Page

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inv-01: Initial Therapy

Covered when ALL of the following are met for initial authorization:

Initial Authorization criteria: Member is ≥1 year of age; confirmed diagnosis of nephropathic cystinosis; prescriber is an endocrinologist, nephrologist, urologist, or other specialist in the treatment of nephropathic cystinosis; diagnosis confirmed by increased leukocyte cystine concentration OR genetic testing showing biallelic pathogenic CTNS variants (submit labs or genetic test results); baseline WBC cystine >2 nmol 1/2 cystine/mg protein (submit labs documenting cystine concentration); serum creatinine <3.0 mg/dL (submit current serum creatinine labs); documented trial and clinically significant intolerance to Cystagon therapy (chart notes submitted; note: frequency of dosing or lack of compliance alone does not constitute intolerance); chart notes documenting current height and weight submitted; member able to take Procysbi on an empty stomach (30 minutes before eating or 2.5 hours after eating); dose will not exceed the maximum FDA-approved dose of 1.95 g/m2 per daysee individual nodes

Initial authorization duration 6 months.

inv-02: Continuation/Reauthorization

Reauthorization covered when ALL of the following are met:

Reauthorization criteria: All initial authorization criteria continue to be met; member has maintained WBC cystine <1 nmol 1/2 cystine/mg protein (submit current labs); chart notes documenting current height and weight submitted; no significant medication-related adverse reactions (eg, GI bleeding, severe skin rashes, seizures, lethargy, somnolence, depression, encephalopathy); serum creatinine remains <3.0 mg/dL and has not increased from baseline (submit current serum creatinine labs).see individual nodes

Reauthorization duration 12 months.

Use of samples to initiate therapy does not meet step-edit/preauthorization criteria. Prior therapies will be verified through pharmacy paid claims or submitted chart notes; documentation that therapy was only provided via samples is not sufficient to satisfy the step-therapy or prior authorization requirement.

Initial Therapy

inv-16: Initial

Initial authorization criteria

Initial: Member is ≥1 year of age.

Initial: Confirmed diagnosis of nephropathic cystinosis (diagnosis confirmed by increased leukocyte cystine concentration OR genetic testing showing biallelic pathogenic CTNS variants; submit labs or genetic test results).

Initial: Prescriber is an endocrinologist, nephrologist, urologist, or other specialist in the treatment of nephropathic cystinosis.

Initial: Baseline WBC cystine >2 nmol 1/2 cystine/mg protein (must submit labs documenting cystine concentration).>2 nmol 1/2 cystine/mg protein

Initial: Serum creatinine <3.0 mg/dL (must submit current serum creatinine lab levels).<3.0 mg/dL

Initial: Trial and clinically significant intolerance to Cystagon therapy documented (chart notes must be submitted).

Frequency of dosing or noncompliance alone is not considered intolerance.

Initial: Chart notes documenting current height and weight submitted.

Initial: Member is able to take Procysbi on an empty stomach (30 minutes before eating or 2.5 hours after eating).

Initial: Member's dose will not exceed the maximum FDA-approved dose of 1.95 g/m2 per day.≤1.95 g/m2 per day

Reauthorization / Continuation Criteria

inv-17: Reauthorization

Reauthorization criteria

Reauthorization: All initial authorization criteria continue to be met.

Reauthorization: Member has maintained WBC cystine <1 nmol 1/2 cystine/mg protein (must submit current lab results).<1 nmol 1/2 cystine/mg protein

Reauthorization: Chart notes documenting current height and weight submitted.

Reauthorization: Member has not experienced any significant medication-related adverse reactions such as gastrointestinal symptoms (GI bleeding, nausea, vomiting, anorexia, or abdominal pain), severe skin rashes, or CNS symptoms (eg, seizures, lethargy, somnolence, depression, encephalopathy).

Reauthorization: Member's serum creatinine is <3.0 mg/dL and has not increased from baseline (must submit current serum creatinine lab levels).<3.0 mg/dL

Diagnosis, Lab Thresholds, and Dose Limits

Diagnosis codingICD-10

ICD Code (if applicable)

ICD code to be provided by prescriber

Baseline WBC cystine

Baseline WBC cystine>2 nmol ½ cystine/mg protein (must submit labs documenting cystine concentration)

Measurement specimenLeukocyte (white blood cell) cystine concentration

Documentation requiredSubmit baseline laboratory results documenting WBC cystine level >2 nmol ½ cystine/mg protein

Reauthorization WBC cystine target

Reauthorization target WBC cystine<1 nmol ½ cystine/mg protein (must submit current lab results)

Required for reauthMember must have maintained WBC cystine <1 nmol ½ cystine/mg protein

Documentation requiredSubmit current laboratory results documenting WBC cystine level

Serum creatinine

Serum creatinine threshold<3.0 mg/dL (must submit current serum creatinine lab levels)

Reauthorization requirementSerum creatinine must remain <3.0 mg/dL and not increased from baseline (must submit current labs)

Documentation requiredSubmit current serum creatinine laboratory results

Maximum dose

Maximum allowable dose≤1.95 g/m2 per day (maximum FDA‑approved dose)

Initial dosing constraintMember's dose will not exceed the maximum FDA‑approved dose of 1.95 g/m2 per day

Documentation recommendedProvide current height and weight to verify mg/m2 dosing calculations

Provider Actions and Documentation Requirements

Prior Authorization

Authorization durations and requirement

Initial authorization is limited to 6 months; reauthorization is limited to 12 months. All listed clinical criteria and supporting documentation must be provided for approval.

Initial Authorization: 6 months
Reauthorization Approval: 12 months

Step Therapy

Previous therapy verification

Previous therapies will be verified through pharmacy paid claims or submitted chart notes. Use of samples does not satisfy step-edit or prior authorization requirements.

Previous therapy verification via pharmacy paid claims or chart notes
Samples do not meet step-edit/preauthorization requirement

Documentation Required

Required supporting documentation

Submit documentation supporting each checked clinical criterion. Required supporting documents include lab results (WBC cystine level, serum creatinine), genetic test results confirming biallelic CTNS pathogenic variants when applicable, and chart notes (including height/weight and documentation of intolerance to prior Cystagon if claimed).

WBC cystine lab results (baseline for initiation; <1 nmol ½ cystine/mg protein for reauthorization)
Serum creatinine lab results (must be <3.0 mg/dL and not increased from baseline for reauthorization)
Genetic test results confirming CTNS biallelic pathogenic variants, if used for diagnosis
Chart notes including height and weight and documentation of intolerance to Cystagon

Documentation Required

Prescriber signature and contact

Prescriber must hand-sign and clearly print their name on the request form; preprinted signature stamps are not acceptable. Prescriber contact information (office contact name, phone, DEA or NPI) must be provided on the request form.

Hand-signed prescriber signature required (preprinted stamps not valid)
Include prescriber contact, office contact name, phone number, and DEA or NPI

Denial Risk

Incomplete or illegible submission

Authorization may be delayed or denied if the request form or clinical information (including labs, diagnostics, or chart notes) is incomplete, incorrect, or illegible. Ensure all required fields on the form are completed and all supporting documentation is legible.

Incomplete or illegible forms and missing clinical documentation can delay or deny authorization
Ensure phone and fax numbers on the form are correct to avoid unnecessary delays

Step	Requirement	Notes
1	Prior therapy must be verified via pharmacy paid claims or submitted chart notes	Use of samples to initiate therapy does not satisfy the step-edit/prior authorization requirement

Background

Nephropathic cystinosis is a genetic disorder caused by pathogenic variants in the CTNS gene that result in accumulation of cystine within cells. Cysteamine therapy reduces intracellular cystine levels to slow disease progression. Procysbi (cysteamine bitartrate) delayed‑release is an FDA‑approved formulation indicated for treatment of nephropathic cystinosis; this policy defines prior authorization and step‑edit requirements for its use, including diagnostic confirmation and laboratory monitoring.

OpenPayer

Procysbi (cysteamine bitartrate) prior authorization

Coverage Criteria for Procysbi (cysteamine bitartrate)

Initial Therapy

Reauthorization / Continuation Criteria

Diagnosis, Lab Thresholds, and Dose Limits

Provider Actions and Documentation Requirements

Step Therapy / Prior Therapy Verification

Site of Care and Specialty Pharmacy

Definitions and Diagnostic Confirmation

Background