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Metabolic Disorders - Cysteamine (Oral) Products for Individual and Family Plans
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Defines prior authorization, coverage criteria, and documentation requirements for Cystagon and Procysbi for members on Cigna Individual and Family Plans for treatment of nephropathic cystinosis.
Criteria of approval were separated for Procysbi and Cystagon; Procysbi criteria now include a minimum age > 1 year.
Genetic confirmation language was clarified to require 'genetic testing confirmed biallelic pathogenic or likely pathogenic variants in the CTNS gene.'
A preferred product table for Individual and Family Plans was added, specifying prior trial/intolerance of Cystagon prior to Procysbi.
Coverage Criteria for Oral Cysteamine Products
Initial FDA-Approved Indication Criteria
Oral cysteamine products are considered medically necessary when ONE of the following is met (I or II):
I. Procysbi (FDA-approved indication)
- Procysbi criteria: A) Patient is > 1 year of age; B) According to the prescriber, diagnosis was confirmed by ONE of the following: i) Genetic testing confirmed biallelic pathogenic or likely pathogenic variants in the CTNS gene [documentation required], OR ii) White blood cell cystine concentration above the upper limit of the normal reference range for the reporting laboratory [documentation required]; C) Patient will not be using Cystagon and Procysbi concurrently; D) The medication is prescribed by or in consultation with a nephrologist or a metabolic disease specialist (or specialist who focuses in the treatment of metabolic diseases); E) Preferred product criteria is met for the product(s) as listed in the Individual and Family Plans table (Procysbi requires prior trial with inadequate efficacy or significant intolerance to Cystagon with documentation).
Documentation required for genetic testing or WBC cystine and for prior trial/intolerance to Cystagon per preferred product table.
II. Cystagon (FDA-approved indication)
- Cystagon criteria: A) According to the prescriber, diagnosis was confirmed by ONE of the following: i) Genetic testing confirmed biallelic pathogenic or likely pathogenic variants in the CTNS gene [documentation required], OR ii) White blood cell cystine concentration above the upper limit of the normal reference range for the reporting laboratory [documentation required]; B) Patient will not be using Cystagon and Procysbi concurrently; C) The medication is prescribed by or in consultation with a nephrologist or a metabolic disease specialist (or specialist who focuses in the treatment of metabolic diseases).
Approval duration 1 year.
Oral cysteamine products are not medically necessary for any indication other than nephropathic cystinosis. This includes all uses outside the FDA‑approved indication for nephropathic cystinosis; the policy will be updated as new published data become available.
The policy specifically states that concomitant therapy with Cystagon and Procysbi is not medically necessary. There are no data to support concurrent use of both oral cysteamine products, and combined therapy is excluded from coverage.
Provider Actions, Prior Authorization, and Documentation
Prior authorization required — 1 year initial approval; nephrologist prescriber or consultation
Prior authorization is required for oral cysteamine products; initial approvals are provided for 1 year. The medication must be prescribed by or in consultation with a nephrologist or a metabolic disease specialist (or specialist who focuses in the treatment of metabolic diseases).
- All approvals are provided for the duration noted (initial approvals = 1 year).
- Because of specialized evaluation and monitoring requirements, initial approval requires the agent to be prescribed by or in consultation with a physician who specializes in the condition being treated.
Step therapy — document prior inadequate efficacy or significant intolerance to Cystagon before Procysbi
For members on Individual and Family Plans seeking Procysbi, the prescriber must document that the patient has tried Cystagon and, according to the prescriber, experienced inadequate efficacy or significant intolerance to Cystagon prior to approval.
- Documentation of the prior trial/intolerance to Cystagon is required.
- Receipt of sample product does not satisfy prior trial requirements.
Prior trial requirement for Procysbi — Cystagon trial and documentation required
Procysbi requires a prior trial with Cystagon demonstrating inadequate efficacy or significant intolerance for coverage under Individual and Family Plans; documentation of that trial and outcome must be provided.
- The policy states: 'Patient has tried, and according to the prescriber, the patient has had inadequate efficacy or significant intolerance to Cystagon... [documentation required]'.
- Documentation may include chart notes, prescription records, and other medical records showing the trial and intolerance or lack of efficacy.
Documentation required — acceptable evidence and required items
Documentation is required where noted and may include chart notes, laboratory tests (e.g., WBC cystine), genetic test results confirming biallelic pathogenic or likely pathogenic CTNS variants, prescription records, and prescriber specialty confirmation.
- Genetic confirmation must be 'genetic testing confirmed biallelic pathogenic or likely pathogenic variants in the CTNS gene' [documentation required].
- WBC cystine concentration above the laboratory reference range is acceptable documentation [documentation required].
- Documentation is required for prior trial/intolerance when seeking Procysbi after Cystagon.
Coding/claim denial risk — missing covered diagnosis or procedure codes will result in denial
Claims submitted without covered diagnosis or procedure codes under the applicable Coverage Policy will be denied as not covered.
- When billing, providers must use the most appropriate codes as of the effective date of the submission.
- Reimbursement is not allowed for services billed for conditions or diagnoses not covered under this Coverage Policy.
Definitions
Background
Nephropathic cystinosis is a rare autosomal recessive lysosomal storage disorder characterized by intracellular cystine accumulation and multisystem organ damage; diagnosis is commonly confirmed by elevated white blood cell (WBC) cystine concentration or molecular testing of the CTNS gene. Prompt initiation of cystine‑depleting therapy is recommended after diagnosis to reduce tissue cystine and slow disease progression.
Oral cysteamine products used to treat nephropathic cystinosis include Cystagon (immediate‑release cysteamine bitartrate) and Procysbi (delayed‑release cysteamine bitartrate). Both agents are approved for the treatment of nephropathic cystinosis; Procysbi’s labeling specifies use in patients aged ≥ 1 year. Treatment must be prescribed by or in consultation with a nephrologist or metabolic disease specialist, and documentation (e.g., WBC cystine or genetic test results) is required to support the diagnosis for coverage decisions.
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