CurrentNeighborhood Health Plan of Rhode IslandPolicy Reference number 2089-A

Cystagon_Medical_Policy

Defines prior authorization criteria, required documentation, and coverage duration for Cystagon (cysteamine bitartrate) for treatment of nephropathic cystinosis. Excludes all non‑FDA or non‑compendial indications as investigational/not medically necessary.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCystagon_Medical_Policy

Policy CodePolicy Reference number 2089-A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit prior authorization with assay showing increased leukocyte cystine concentration or genetic testing for initial requests and lab results or chart notes documenting clinical response for continuation requests.

POLICY UPDATE CHANGES

No material clinical or coverage changes

1Covered Indications (FDA‑approved)

All other indicationsNot covered (experimental/investigational)

12 monthsAuthorization duration (initial/continuation)

Coverage Summary

Scope: Defines prior authorization criteria, required documentation, and coverage duration for Cystagon (cysteamine bitartrate) for treatment of nephropathic cystinosis. Coverage stance: covered_with_criteria. Authorization duration: 12 months. FDA indication: Cystagon is indicated for the management of nephropathic cystinosis in children and adults per the package labeling. Reference number 2089-A.

Initial Therapy Criteria

Prior Authorization

Prior authorization required

Initial and continuation requests require prior authorization; initial requests must include assay showing increased leukocyte cystine concentration or genetic testing confirming diagnosis; continuation requests must include lab results or chart notes documenting clinical response (improvement, stabilization, or slowing of disease progression).

Documentation Required

Required documentation for initial requests

Submit assay detecting increased cystine concentration in leukocytes or genetic testing results supporting diagnosis.

Continuation Therapy Criteria (Reauthorization)

Documentation Required

Required documentation for continuation requests

Submit lab results or chart notes documenting a positive response to therapy (e.g., improvement, stabilization, or slowing of disease progression for serum creatinine, calculated creatinine clearance, leukocyte cystine concentration, or maintained growth).

Unproven / Exclusions

Non‑Covered Indications

All other indications (non‑FDA‑approved and non‑compendial uses) are considered experimental/investigational and not medically necessary

Billing Rule

Combination therapy exclusion

Cystagon will not be authorized for members who will use it in combination with Procysbi.

Provider Actions / Documentation Requirements

Prior Authorization

Prior authorization required

Initial and continuation requests require prior authorization. Authorization may be granted for up to 12 months for both initial treatment and continuation of therapy when criteria are met.

Documentation Required

Required documentation for initial requests

Submit assay detecting increased cystine concentration in leukocytes or genetic testing results supporting diagnosis.

Documentation Required

Required documentation for continuation requests

Billing Rule

Combination therapy exclusion

Cystagon will not be authorized for members who will use it in combination with Procysbi.

Background

Background: Cystagon (cysteamine bitartrate) is indicated for management of nephropathic cystinosis in children and adults per FDA labeling. The policy requires objective confirmation of diagnosis for initial authorization (assay showing increased leukocyte cystine concentration or genetic testing) and requires evidence of clinical response for continued therapy (documentation of improvement, stabilization, or slowing of disease progression for serum creatinine, calculated creatinine clearance, or leukocyte cystine concentration). Nephropathic cystinosis confirmation is defined as the presence of increased cystine concentration in leukocytes or genetic testing consistent with cystinosis.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCystagon_Medical_Policy

Policy CodePolicy Reference number 2089-A

Change TypeNo material change

Effective Date

Next Review Date

On This Page

OpenPayer

Cystagon_Medical_Policy

Coverage Summary

Initial Therapy Criteria

Continuation Therapy Criteria (Reauthorization)

Unproven / Exclusions

Applicable Codes

Provider Actions / Documentation Requirements

Clinical Evidence

Background

Revision History