CurrentCentenePolicy CP.PHAR.155

Cysteamine oral (Cystagon, Procysbi) coverage

Defines medical necessity criteria, prior authorization requirements, and approval durations for oral cysteamine (Cystagon, Procysbi) for members covered by Centene lines of business including Commercial, HIM/ICHRA, and Medicaid.

Policy Summary

PayerCentene

PolicyCysteamine oral (Cystagon, Procysbi) coverage

Policy CodePolicy CP.PHAR.155

Change TypeMaterial updates and additions

Effective Date

Next Review Date

Key ActionObtain prior authorization and submit documentation confirming diagnosis, prescriber specialty, prior use when required, response for continued therapy, and dosing within limits.

SourceLink

POLICY UPDATE CHANGES

Added prescriber requirement for nephrologist or a metabolic disease specialist experienced in management of nephropathic cystinosis (e.g., endocrinologist or urologist).

Added step therapy bypass for Illinois HIM per IL HB 5395.

For initial therapy, approval duration revised from 6 months to 12 months.

Added ICHRA (individual coverage health reimbursement arrangement) as a line of business.

12 monthsapproval duration

1.95 g/m2/daymaximum daily dose

Procysbi ≥1 yrFDA age indication

Commercial, HIM/ICHRA, Medicaid

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Coverage Criteria for Oral Cysteamine

Initial Therapy

Covered when ALL of the following are met:

Initial Nephropathic Cystinosis Criteria: 1. Diagnosis of nephropathic cystinosis confirmed by one of the following: (a) increased leukocyte cystine concentration above the upper limit of the normal reference range for the reporting laboratory; OR (b) pathogenic CTNS gene mutation; OR (c) corneal crystals on slit lamp examination; 2. Prescribed by or in consultation with a nephrologist or a metabolic disease specialist experienced in management of nephropathic cystinosis (e.g., endocrinologist or urologist); 3. If Procysbi is requested, member must have used Cystagon unless contraindicated or clinically significant adverse effects are experienced; (Illinois HIM requests are exempt from the step therapy requirement per IL HB 5395); 4. Request is not for combination use of Procysbi and Cystagon; 5. Dose does not exceed 1.95 g per m2 per day.dose <= 1.95 g/m2/day

Approval duration: 12 months

Continuation Therapy

Covered when ALL of the following are met:

Continued Nephropathic Cystinosis Criteria: 1. Member meets one of the following: (a) currently receiving medication via Centene benefit or previously met initial approval criteria; OR (b) currently receiving medication and enrolled in a state/product with continuity of care regulations; 2. Member is responding positively to therapy as evidenced by improvement in leukocyte cystine concentration within the past 3 months; 3. If request is for a dose increase, the new dose does not exceed 1.95 g per m2 per day.dose <= 1.95 g/m2/day

Approval duration: 12 months

Other Indications

Covered when ONE of the following applies:

Other Indications - recent label change: If this drug has had a label change within the last 6 months that is not yet reflected in this policy (e.g., newly approved indication, age expansion, new dosing regimen), follow the applicable formulary or non-formulary policy for the relevant line of business as specified (see policy references).

Refer to CP.CPA.190, HIM.PA.33/103, CP.PMN.255/16 as applicable

Other Indications - off-label process: If the requested use (diagnosis, age, dosing regimen) is not specifically listed under 'Diagnoses/Indications for which coverage is NOT authorized' AND criterion 1 above does not apply, refer to the off-label use policy for the relevant line of business (CP.CPA.09, HIM.PA.154, CP.PMN.53).

Refer to off-label policies

Non‑FDA approved indications for oral cysteamine (Cystagon, Procysbi) are not authorized unless the request includes sufficient documentation of efficacy and safety consistent with the applicable off‑label use policy or evidence of coverage documents (see CP.CPA.09 for Commercial, HIM.PA.154 for HIM/ICHRA, and CP.PMN.53 for Medicaid).

Uses for indications that are not FDA‑approved and that are not supported by the referenced off‑label use policy or evidence of coverage are considered not authorized for coverage.

Coding and Dose Limits

Neutral / product codesmixed

NDC	NDCs not listed in policy

inv-07: Maximum daily dose — ≤ 1.95 g/m2/day.

Maximum daily dose≤ 1.95 g/m2/day

Source guidancePolicy dosing statement: 'Dose does not exceed 1.95 g per m2 per day.'

Drug-specific maximums (per dosing table)Cystagon and Procysbi maximum listed as 1.95 g/m2/day in Dosage and Administration.

Prior Authorization, Step Therapy, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization required and approved for 12 months when criteria are met. Documentation must confirm that the member meets all approval criteria (diagnosis confirmation, prescriber specialty, step therapy when applicable).

Approval duration: 12 months

Step Therapy

Step Therapy Requirement for Procysbi

Member must use Cystagon before Procysbi unless contraindicated or clinically significant adverse effects are experienced. For Illinois HIM requests, the step therapy requirement does not apply as of 1/1/2026 per IL HB 5395.

Documentation Required

Definitions and Laboratory Targets

inv-13: Leukocyte (WBC) cystine concentration — Lab measurement used to confirm diagnosis and monitor response; therapy goal described.

DefinitionLeukocyte (WBC) cystine concentration is a laboratory measurement of intracellular cystine used to confirm diagnosis and monitor response.

Diagnostic roleAn increased leukocyte cystine concentration above the laboratory upper limit of normal is one criterion to confirm nephropathic cystinosis.

Therapeutic goalThe goal of cysteamine therapy is to lower WBC cystine levels (used to monitor response).

Assay variabilityReported values vary by assay method and units (e.g., nmol half‑cystine/mg protein); results from different methods may not be interchangeable.

inv-14: Maximum dose — Dose should not exceed 1.95 g per m2 per day.

Maximum dose limit

Clinical Background

Nephropathic cystinosis is treated with cysteamine, a cystine‑depleting agent; the therapeutic goal of therapy is to lower leukocyte (WBC) cystine concentration to demonstrate biochemical response and guide treatment.