CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Medicaid_Ztalmy_20250820

Defines coverage and prior authorization criteria for Ztalmy (ganaxolone) oral suspension for treatment of seizures associated with CDKL5 deficiency disorder (CDD) for patients aged 2 years and older, including initial and continuation criteria and quantity limits.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyMedicaid_Ztalmy_20250820

Policy CodePolicy N/A

Change TypeReviewed (no clinical changes)

Effective DateMar 1, 2023

Next Review Date

Key ActionSubmit documentation showing age ≥2 years, neurologist prescribing or consultation, dosing within specified weight-based limits, genetic testing showing pathogenic/likely pathogenic CDKL5 mutation, and baseline monthly seizure frequency to obtain a 6-month authorization.

SourceLink

POLICY UPDATE CHANGES

Reviewed: 12/22, 06/23, 6/24, 8/25 (no clinical policy statement changes indicated).

1Covered Indications (FDA-approved)

6 monthsInitial Authorization Duration

10 bottles/30 daysQuantity Limit

>=2 yrsMinimum Age

Scope: Ztalmy (ganaxolone) oral suspension is covered only for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged ≥ 2 years. Other uses of Ztalmy are considered experimental/investigational and are not covered. Effective date: 2023-03-01. Last review: 2025-08-25.

Key Policy Thresholds

Minimum Age>= 2 years

Weight-based dosing threshold<= 28 kg: 63 mg/kg/day; > 28 kg: <= 1,800 mg/day

Initial authorization duration6 months

Continuation authorization duration6 months

Stable maintenance dose prior to renewal>= 3 months

Quantity limit10 bottles (110 mL each) per 30 days (36.667 mL/day)

Initial Therapy Criteria

An authorization may be granted for 6 months when ALL of the following criteria are met:

ALL of the following

The patient is 2 years of age or older>= 2 years
Ztalmy (ganaxolone) is prescribed by, or in consultation with, a neurologist
Prescriber specialty must be documented in chart notes or referral/consultation documentation
Dosing limits
- Weight ≤ 28 kg: total daily dose does not exceed 63 mg/kg per day<= 63 mg/kg/day
  Dosing must be weight-based and documented
- Weight > 28 kg: total daily dose does not exceed 1,800 mg per day<= 1800 mg/day
  Dosing must be documented
Required clinical documentation
- Genetic testing demonstrating a pathogenic or likely pathogenic mutation in the CDKL5 gene (e.g., genetic test report or chart documentation of result)
  Enzyme assay or genetic testing documentation acceptable; report must be available
- Baseline monthly seizure frequency documented in chart notes or medical record
  Must include seizure type and frequency prior to initiation of therapy

Prior Authorization

Prior Authorization Required for Initial Therapy

Prior authorization is required for initial therapy. Submit documentation showing age, neurologist prescribing or consultation, dosing within limits, genetic test demonstrating pathogenic/likely pathogenic CDKL5 mutation, and baseline monthly seizure frequency. Initial approvals are for 6 months.

Member age (proof of DOB)
Prescriber specialty or consultation note from a neurologist
Weight and dosing calculation showing total daily dose within limits (63 mg/kg/day if ≤28 kg; ≤1,800 mg/day if >28 kg)
Genetic testing report or chart documentation confirming pathogenic/likely pathogenic CDKL5 mutation
Baseline monthly seizure frequency with seizure types (chart notes or medical record)
Note: Initial authorization duration = 6 months

Continuation Therapy Criteria

Prior Authorization

Prior authorization for continuation

For renewal/continuation, provide clinical documentation that the patient has been on a stable maintenance dose for at least 3 months and has demonstrated clinical benefit either by decreased seizures or improved quality of life on a validated scale. Renewals may be granted for 6 months.

Statement of stable maintenance dosing for ≥3 months (medication administration records or clinician note)
Evidence of response examples: seizure frequency logs showing reduction in monthly seizures; clinician progress notes documenting decreased seizure burden; scores from validated QoL instruments demonstrating improvement

Unproven / Exclusions

Not Covered

All other indications are considered experimental/investigational and are not a covered benefit.

Use of Ztalmy for indications other than seizures associated with CDKL5 deficiency disorder (CDD) is not covered (experimental/investigational)

Denial Risk

Off-label uses are not covered (denial risk)

Uses of Ztalmy outside the FDA-approved indication for seizures associated with CDKL5 deficiency disorder are considered experimental/investigational and will be denied. Ensure the indication is documented — e.g., CDKL5 diagnosis supported by genetic testing or enzyme assay in the medical record.

Document the indication: diagnosis of seizures due to CDKL5 deficiency disorder
Provide genetic testing or enzyme assay demonstrating pathogenic/likely pathogenic CDKL5 mutation

Applicable Codes

Drug formulation referencedNDC

oral suspension 50 mg/mL

Ztalmy (ganaxolone) oral suspension concentration referenced for quantity limits

Billing Rule

Quantity limit enforcement

Quantity limit: coverage is limited to 10 bottles (110 mL total) of Ztalmy oral suspension 50 mg/mL per 30 days (which equals 36.667 mL per day).

Formulation referenced: Ztalmy oral suspension 50 mg/mL
10 bottles per 30 days = 110 mL total; 110 mL / 30 days = 36.667 mL/day (supply mapping to days/supplies)

Provider Actions & Documentation

Documentation Required

Genetic test documentation required

Provide enzyme assay or genetic testing results in the chart notes or medical record demonstrating a pathogenic or likely pathogenic mutation in the CDKL5 gene as part of the authorization request.

Acceptable documentation: chart notes with genetic test interpretation, laboratory genetic test reports, enzyme assay reports
Genetic counseling notes or summaries placed in the medical record that document the pathogenic/likely pathogenic CDKL5 mutation

Operational notes: Ztalmy must be prescribed by, or in consultation with, a neurologist. Dosing thresholds must be documented in the medical record, including patient weight and the dosing calculation to demonstrate compliance with the weight-based limits (≤ 28 kg: 63 mg/kg/day; > 28 kg: 1,800 mg/day).

Background

Background: Ztalmy (ganaxolone) is FDA-approved for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in patients 2 years of age and older. This policy limits coverage to that FDA-approved indication; all other uses are considered experimental/investigational and are not a covered benefit.

Definitions: CDD = Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder.