CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Cystagon 2089 A Sgm P2022

Policy governs prior authorization, documentation, initial and continuation criteria for Cystagon for the management of nephropathic cystinosis; all other indications are considered experimental/investigational and not medically necessary.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCystagon 2089 A Sgm P2022

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit prior authorization with required documentation: for initial requests, assay showing increased leukocyte cystine concentration or genetic testing results; for continuation requests, lab results or chart notes documenting response to therapy.

SourceLink

POLICY UPDATE CHANGES

No material changes — policy remains current with existing coverage criteria and documentation requirements.

1Covered Indication (nephropathic cystinosis)

All othersNot covered indications (experimental/investigational)

12Authorization duration (months)

Coverage Summary

Covered with criteria: Cystagon (cysteamine) is covered for the FDA‑approved indication nephropathic cystinosis when the policy's prior authorization criteria are met. Authorization duration: 12 months. Initial approval requires diagnostic confirmation (see required documentation). For continuation, documentation of clinical response is required and reauthorization may be granted for 12 months. All other indications beyond nephropathic cystinosis are considered experimental/investigational and not medically necessary.

Initial Therapy Criteria - Nephropathic Cystinosis

Initial Authorization - Nephropathic Cystinosis

Authorization of 12 months may be granted...

ALL of the following

Diagnosis of cystinosis confirmed by the presence of increased cystine concentration in leukocytes or by genetic testing
Required documentation for initial requests: assay detecting increased cystine concentration in leukocytes or genetic testing results supporting diagnosis.
Member will not use Cystagon in combination with Procysbi
Concomitant use with Procysbi is prohibited.

Continuation / Reauthorization Criteria - Nephropathic Cystinosis

Continuation/Reauthorization - Nephropathic Cystinosis

Authorization of 12 months may be granted for continued treatment when ALL of the following are met:

ALL of the following

Member has diagnosis of nephropathic cystinosis (established previously per initial criteria)
Continuation requests require prior diagnosis confirmation on initial request.
ALL of the following
- Evidence of response (any)
  - Improvement, stabilization, or slowing of disease progression for serum creatinine
  - Improvement, stabilization, or slowing of disease progression for calculated creatinine clearance
  - Improvement, stabilization, or slowing of disease progression in leukocyte cystine concentration
  - Maintained growth (height)
    Mentioned as example in documentation section
No concurrent use of Procysbi
Same prohibition as for initial therapy.

Provider Actions

Prior Authorization

Prior authorization required

Submit a prior authorization (PA) request with the required documentation for both initial and continuation therapy. For initial requests include diagnostic proof (assay detecting increased leukocyte cystine concentration or genetic testing results). For continuation requests include objective evidence of clinical benefit (lab results or chart notes) to support reauthorization.

Initial: assay detecting increased cystine concentration in leukocytes OR genetic testing results supporting diagnosis
Continuation: lab results or chart notes documenting response to therapy (e.g., improvement, stabilization, or slowing of disease progression)

Documentation Required

Document evidence of response for reauthorization

Provide documentation of clinical response when requesting reauthorization. Include relevant laboratory values or clinical notes demonstrating improvement, stabilization, or slowing of disease progression for renal function and cystine burden, or evidence of maintained growth.

Serum creatinine values showing improvement/stabilization/slowing of progression
Calculated creatinine clearance showing improvement/stabilization/slowing of progression
Leukocyte cystine concentration showing improvement/stabilization/slowing of progression
Evidence of maintained growth (height) as applicable

Denial Risk

Concomitant therapy exclusion

Claims may be denied or authorization may not be granted if Cystagon is used concomitantly with Procysbi. Confirm the member will not use Cystagon in combination with Procysbi before approving or submitting claims.

Clinical Evidence & References

Reference: Cystagon [package insert]. Mylan Pharmaceuticals Inc.; January 2019.

Clinical background: Cystagon (cysteamine) is FDA‑approved for the management of nephropathic cystinosis in children and adults. The policy requires diagnostic confirmation (e.g., increased leukocyte cystine concentration or genetic testing) for initial authorization and documentation of response for continuation. Uses of Cystagon outside the management of nephropathic cystinosis are considered investigational.