CurrentMass General Brigham Health PlanPolicy N/A

Ztalmy (ganaxolone) — Coverage Criteria for CDKL5 Deficiency Disorder Seizures

Policy governing prior authorization, specialty pharmacy designation, and coverage criteria for Ztalmy (ganaxolone) to treat seizures associated with CDKL5 deficiency disorder (CDD) for Mass General Brigham Health Plan members.

Policy Summary

PayerMass General Brigham Health Plan

PolicyZtalmy (ganaxolone) — Coverage Criteria for CDKL5 deficiency disorder seizures

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateMar 1, 2024

Next Review DateN/A

Key ActionObtain prior authorization and submit documentation of pathogenic CDKL5 mutation, diagnosis of CDD, and neurologist involvement.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2 yrsminimum patient age

6 moinitial auth

12 moreauthorization

Specialtyfill type

Coverage Criteria for Ztalmy (ganaxolone)

ALL of the following

The member has a documented diagnosis of cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD).
Medical records supporting the diagnosis with enzyme assay or genetic testing demonstrating a pathogenic or likely pathogenic mutation in the CDKL5 gene.
The medication is prescribed by or in consultation with a neurologist.
For members new to the plan, the member is currently receiving treatment with the requested medication. (Products obtained as samples or via the manufacturer's patient assistance programs do NOT qualify as 'currently receiving' for continued authorization.)

Continuation Therapy

Covered when ALL of the following are met:

ALL of the following

The member has an established diagnosis of CDKL5 deficiency disorder (CDD).
The member has achieved or maintained a positive clinical response to therapy, such as a decrease in seizure frequency or severity while on treatment.
The prescribing clinician documents continued benefit and plans for ongoing monitoring and follow-up.

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required. Authorization may be granted for members new to the plan who are currently receiving the requested medication (authorization excludes product obtained as samples or via manufacturer's patient assistance programs). Authorizations for members new to the plan who are not currently receiving the medication may be denied (exceptions apply for certain acquisition routes).

May be denied for new-to-plan members not currently receiving the medication (exceptions for certain acquisition routes)

Billing Rule

Specialty Pharmacy Requirement

This medication is designated as a specialty medication and must be filled at a contracted specialty pharmacy.

Specialty pharmacy contact (All Plans): Phone: 877-519-1908
Non-specialty medications contact (All Plans): Phone: 800-711-4555; Fax: 844-403-1029

Documentation Required

Required Clinical Documentation

Medical records documenting genetic testing demonstrating a pathogenic CDKL5 mutation and diagnostic documentation (e.g., diagnosis of CDKL5 deficiency disorder and documentation that medication is prescribed by or in consultation with a neurologist) should be provided to support authorization.

Medical records demonstrating genetic testing/pathogenic CDKL5 mutation and diagnostic documentation
Prescription by or consultation with a neurologist

Background

Ztalmy (ganaxolone) is indicated for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in patients aged 2 years and older. Continuation of therapy may be authorized when the member with CDD achieves or maintains a positive clinical response, such as a reduction in seizure frequency.

Definitions

Ztalmy — Ganaxolone

Generic nameGanaxolone

Brand name / formulationZtalmy

FDA‑indicated useTreatment of seizures associated with CDKL5 deficiency disorder (CDD)

Minimum patient age for indication2 years and older

Documented evidence sourceManufacturer/label overview specifying indication and age

CDKL5 deficiency disorder (CDD)

Disease nameCDKL5 deficiency disorder (CDD)

Genetic causePathogenic mutations in the CDKL5 (cyclin‑dependent kinase‑like 5) gene

Key clinical featureAssociated with treatment‑resistant seizures

Documentation required for coverageMedical records documenting enzyme assay or genetic testing demonstrating a pathogenic CDKL5 mutation

Prescriber requirementMedication prescribed by or in consultation with a neurologist (per authorization criteria)