CurrentNorth Carolina MedicaidPolicy N/A

Ctexli Coverage for Cerebrotendinous Xanthomatosis

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This policy governs North Carolina Medicaid prior approval for Ctexli, a bile therapy used to treat cerebrotendinous xanthomatosis. It applies to eligible Medicaid beneficiaries and specifies diagnostic, specialist-prescribing, monitoring, continuation, and authorization-duration requirements.

Policy Summary

PayerNorth Carolina Medicaid

PolicyCtexli Coverage for Cerebrotendinous Xanthomatosis

Policy CodePolicy N/A

Change TypeNo material change

Effective DateNovember 5, 2026

Next Review DateN/A

Key ActionCtexli requires prior approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

18+Minimum age for initial approval

6 monthsMaximum authorization period

2Accepted diagnostic pathways

AnnualLiver monitoring frequency

Required

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CurrentNorth Carolina MedicaidPolicy N/A

Ctexli Coverage for Cerebrotendinous Xanthomatosis

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Policy Summary

PayerNorth Carolina Medicaid

PolicyCtexli Coverage for Cerebrotendinous Xanthomatosis

Policy CodePolicy N/A

Change TypeNo material change

Effective DateNovember 5, 2026

Next Review DateN/A

Key ActionCtexli requires prior approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

18+Minimum age for initial approval

6 monthsMaximum authorization period

2Accepted diagnostic pathways

AnnualLiver monitoring frequency

Required

Continuation response

Coverage Criteria

Initial Therapy

Covered for initial therapy when all of the following are met.

Initial therapy requirements

Age: The beneficiary is 18 years of age or older.>= 18 years
ONE of: diagnostic confirmation
- Genetic testing: Molecular genetic testing shows a pathogenic variant in the CYP27A1 gene.Pathogenic CYP27A1 variant
- Laboratory testing shows elevated serum cholestanol levels.Elevated serum cholestanol
Specialist prescribing: Ctexli is prescribed by or in consultation with a specialist who treats patients with CTX, such as a geneticist, neurologist, ophthalmologist, or metabolic specialist.
Concurrent Chenodal restriction: Ctexli is not prescribed concurrently with Chenodal.No concurrent Chenodal
Bile acid salts: Ctexli is not prescribed concurrently with bile acid salts unless clinically appropriate.Concurrent use permitted only when clinically appropriate
Monitoring agreement: The prescriber agrees to monitor ALT, AST, and bilirubin levels annually or as clinically indicated.Annual or clinically indicated monitoring
Baseline laboratory testing: Baseline liver transaminase ALT and AST and total bilirubin levels are obtained before initiating Ctexli treatment.Before treatment initiation

Continuation Therapy

Covered for continuation when all of the following are met.

Continuation therapy requirements

Positive response: Documentation shows a positive response to therapy, such as decreased or stabilized bile alcohol levels, reduced signs and symptoms of CTX, or reduced plasma cholestanol levels.Decreased or stabilized bile alcohol levels, reduced CTX signs or symptoms, or reduced plasma cholestanol levels
Tolerability: The beneficiary has not experienced unacceptable toxicity or treatment-restricting adverse effects from the medication.No unacceptable toxicity or treatment-restricting adverse effects

Concurrent prescribing of Ctexli with Chenodal is excluded from approval. Ctexli must not be prescribed at the same time as Chenodal.

Provider Actions and Authorization Requirements

Prior Authorization

Prior Approval and EPSDT Requirements

Prior approval is required whenever the service, product, or procedure requires it. Beneficiaries under 21 are not exempt from prior approval requirements under EPSDT; consult the NCTracks Provider Claims and Billing Assistance Guide and the EPSDT provider page for additional requirements.

Do not treat EPSDT eligibility as eliminating a required prior approval.
Follow applicable NCTracks and EPSDT provider guidance.

Prior Authorization

Authorization Duration and PDL Criteria

Ctexli authorization may be approved for up to 6 months. If the requested medication is on the Preferred Drug List (PDL), the applicable PDL criteria also apply.

Authorization duration: up to 6 months.
Apply PDL requirements when the requested medication is listed on the PDL.

Background

Cerebrotendinous xanthomatosis (CTX) is the condition evaluated for Ctexli coverage under this policy. Coverage requires confirmation through either a pathogenic CYP27A1 variant identified by molecular genetic testing or elevated serum cholestanol levels. Ctexli therapy also requires baseline liver testing and ongoing monitoring of ALT, AST, and bilirubin, with continuation dependent on documented clinical or biochemical response and acceptable tolerability.

Definitions

Confirmed cerebrotendinous xanthomatosis (CTX)

Molecular confirmationMolecular genetic testing shows a pathogenic variant in the CYP27A1 gene.

Laboratory confirmationLaboratory testing shows elevated serum cholestanol levels.

Diagnostic requirementOne of the two accepted confirmation pathways must establish the diagnosis of cerebrotendinous xanthomatosis (CTX).

EPSDT

MeaningEarly and Periodic Screening, Diagnostic, and Treatment (EPSDT) is a federal Medicaid requirement for medically necessary services, products, or procedures for beneficiaries under 21 years of age.

PurposeCoverage applies when the service is medically necessary to correct or ameliorate a defect, physical or mental illness, or condition identified through a screening examination.

Ctexli Initial Therapy

Initial approval requires all of the following, including one of the two accepted diagnostic confirmation pathways.

Ctexli initial therapy

Age: The beneficiary is 18 years of age or older.>= 18 years
ONE of: diagnostic confirmation
- Genetic confirmation: A molecular genetic test shows a pathogenic variant in the CYP27A1 gene.Pathogenic CYP27A1 variant
- Laboratory confirmation:

Ctexli Continuation Therapy

Continuation approval requires all of the following.

Ctexli continuation therapy

Positive response: Documentation shows a positive response to therapy, such as decreased or stabilized bile alcohol levels, reduced signs and symptoms of CTX, or reduced plasma cholestanol levels.Decreased or stabilized bile alcohol levels, reduced CTX signs or symptoms, or reduced plasma cholestanol levels
Safety and tolerability: The beneficiary has not experienced unacceptable toxicity or treatment-restricting adverse effects from the medication.No unacceptable toxicity or treatment-restricting adverse effects

Preferred Drug List Step Therapy

PDL pathway	Requirement
Preferred product trial	Try and fail the preferred product(s) first.
Clinical exception	Document a clinical reason the preferred product(s) cannot be tried.
Other PDL criteria	Meet other applicable Preferred Drug List criteria, as specified on the PDL.

Policy Summary

PayerNorth Carolina Medicaid

PolicyCtexli Coverage for Cerebrotendinous Xanthomatosis

Policy CodePolicy N/A

Change TypeNo material change

Effective DateNovember 5, 2026

Next Review DateN/A

Key ActionCtexli requires prior approval.

SourceLink

On This Page

Step Therapy

Preferred Drug List Requirements

Before requesting Ctexli, document that applicable preferred-product requirements have been addressed. The request must show a trial and failure of the preferred product(s), or a clinical reason the preferred product(s) cannot be tried, along with any other criteria specified on the PDL.

Document try-and-fail of preferred product(s) when required.
If preferred product(s) cannot be tried, document the clinical reason.
Address any additional PDL criteria applicable to the request.

Documentation Required

Initial Diagnostic and Monitoring Documentation

For initial approval, document the required diagnosis, specialist involvement, treatment exclusions, and laboratory monitoring plan. CTX must be confirmed by either a molecular genetic test showing a pathogenic CYP27A1 variant or elevated serum cholestanol levels.

Beneficiary must be 18 years of age or older.
Ctexli must be prescribed by or in consultation with a specialist who treats CTX, such as a geneticist, neurologist, ophthalmologist, or metabolic specialist.
Document that Ctexli is not being prescribed concurrently with Chenodal.
Document that Ctexli is not being prescribed concurrently with bile acid salts unless clinically appropriate.
Obtain baseline ALT, AST, and total bilirubin before initiating treatment.
Prescriber must agree to monitor ALT, AST, and bilirubin annually or as clinically indicated.

Denial Risk

Initial Approval Denial Conditions

Initial approval should be denied when the beneficiary does not meet all applicable initial criteria. This includes failure to establish CTX, failure to meet the age or specialist requirements, prohibited concurrent therapy, or failure to provide required baseline laboratory documentation and monitoring agreement.

No qualifying CTX confirmation by CYP27A1 molecular genetic testing or elevated serum cholestanol.
Beneficiary is under 18 years of age.
Required specialist prescribing or consultation is not documented.
Concurrent Chenodal use or non-clinically appropriate concurrent bile acid salt use is identified.
Baseline ALT, AST, and total bilirubin results are missing, or the prescriber does not agree to required monitoring.

Documentation Required

Continuation Response Documentation

For continuation approval, document a positive clinical or laboratory response to therapy and confirm that treatment has not caused unacceptable toxicity or treatment-restricting adverse effects.

Examples of response include decreased or stabilized bile alcohol levels, reduced CTX signs and symptoms, or reduced plasma cholestanol levels.
Document that unacceptable toxicity or treatment-restricting adverse effects have not occurred.

Denial Risk

Continuation Denial Conditions

Continuation should be denied when the record does not demonstrate a positive response to Ctexli or when the beneficiary has experienced unacceptable toxicity or treatment-restricting adverse effects.

Insufficient clinical or laboratory response is a continuation denial condition.
Unacceptable toxicity or treatment-restricting adverse effects are continuation denial conditions.

Denial Risk

PDL Requirements Not Met

If applicable PDL step-therapy requirements are not met, the request may be denied or additional documentation may be required. Ensure the preferred-product trial, failure, clinical exception, and any other PDL criteria are clearly documented.

Missing documentation of try-and-fail with the preferred product(s) may result in denial or a request for more information.
If preferred product(s) cannot be tried, document the clinical reason.
Failure to satisfy other applicable PDL criteria creates additional denial risk.

Policy limitsClinical policy limitations may be exceeded or may not apply when provider documentation demonstrates that the requested service meets all EPSDT medical-necessity criteria.

Prior approvalEPSDT does not eliminate a prior approval requirement when the service, product, or procedure requires prior approval.

Serum cholestanol levels are elevated.

Elevated serum cholestanol

Specialist prescribing or consultation: Ctexli is prescribed by or in consultation with a specialist who treats patients with CTX, such as a geneticist, neurologist, ophthalmologist, or metabolic specialist.

Concomitant therapy restrictions: Ctexli is not prescribed concurrently with Chenodal and is not prescribed concurrently with bile acid salts unless clinically appropriate.No concurrent Chenodal; bile acid salts only when clinically appropriate

Laboratory monitoring: Baseline ALT, AST, and total bilirubin are obtained before treatment, and the prescriber agrees to monitor these levels annually or as clinically indicated.Baseline before initiation; annual or clinically indicated thereafter