CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Waskyra (etuvetidigene autotemcel) Coverage Criteria

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This policy governs prior authorization and coverage of Waskyra for eligible pediatric patients and adults with Wiskott-Aldrich Syndrome under the New Hampshire Medicaid Fee-for-Service Program. Coverage requires all clinical, transplant eligibility, safety, monitoring, and treatment-use criteria to be satisfied.

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicyWaskyra (etuvetidigene autotemcel) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation demonstrating that all 16 Waskyra approval criteria are satisfied.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 moMinimum patient age

16Approval criteria

>5 g/LSerum IgG minimum

NegativeRequired HIV status

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CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Waskyra (etuvetidigene autotemcel) Coverage Criteria

Customize your policy alerts

Sign up for all New Hampshire Department of Health and Human Services policy alerts

Know when New Hampshire Department of Health and Human Services releases new policies or updates existing guidance.

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Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicyWaskyra (etuvetidigene autotemcel) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation demonstrating that all 16 Waskyra approval criteria are satisfied.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 moMinimum patient age

16Approval criteria

>5 g/LSerum IgG minimum

NegativeRequired HIV status

Contraception period

All requiredCoverage stance

Coverage Criteria

Initial Therapy Criteria

Covered when all of the following are met.

Waskyra initial therapy criteria

Age: Patient is 6 months of age or older.6 months or older
Diagnosis: Patient has a confirmed diagnosis of Wiskott-Aldrich Syndrome as confirmed by molecular genetic testing and has a severe phenotype WAS mutation.Confirmed molecular diagnosis with severe phenotype WAS mutation
Donor availability: Patient does not have a medically eligible 10/10 HLA-identical matched stem cell donor for transplant.No medically eligible 10/10 HLA-identical matched donor
Requests may be reviewed case by case when an HLA-identical donor is identified but mobilized peripheral blood or bone marrow stem-cell collection is not feasible.
Prior HSCT and autologous candidacy: Patient has not received a prior allogeneic HSCT within 6 months before screening, or, if such a transplant occurred, has no evidence of residual donor cells, and is a candidate for autologous stem cell transplantation with adequate renal and hepatic function as applicable.No qualifying recent allogeneic HSCT or no residual donor cells; suitable for autologous transplantation
Treatment use: Waskyra is used as single-agent therapy.Single agent
This requirement does not apply to lymphodepleting or conditioning therapy or pre-emptive anti-CD20 therapy while awaiting manufacture.
Prior gene therapy: Patient has not received prior hematopoietic stem cell-based gene therapy.No prior hematopoietic stem cell-based gene therapy
Hypersensitivity: Patient does not have a history of hypersensitivity to dimethyl sulfoxide, the active substance of Waskyra, or any excipient.No relevant hypersensitivity history
HIV status: Patient is HIV negative as confirmed by a negative HIV test before mobilization.Negative HIV test before mobilization
Veno-occlusive disease monitoring: Patient will be monitored for signs and symptoms of veno-occlusive disease, including liver function tests, during the first month after infusion.Monitoring during first month after infusion
Infection monitoring and prophylaxis: Patient will be monitored for signs and symptoms of serious infection and will receive prophylactic antimicrobials according to standard local or institutional guidelines.Monitoring and prophylactic antimicrobials
Immunoglobulin G: Patient's serum immunoglobulin G level is above 5 g/L.>5 g/L
Malignancy monitoring: Patient will be monitored periodically for malignancies after treatment.Periodic post-treatment monitoring
Stem-cell mobilization: Patient will undergo stem-cell mobilization using G-CSF and plerixafor according to established protocols.G-CSF plus plerixafor
Warnings and interactions: Prescriber has reviewed Waskyra warnings, precautions, and drug interactions and will monitor patient status as appropriate.Prescriber review and monitoring
Pregnancy testing: Females of reproductive potential have a negative serum pregnancy test before mobilization, with repeat confirmation before conditioning procedures and before Waskyra administration.Negative serum pregnancy tests at required time points
Contraception: Males capable of fathering a child and females of childbearing age use an effective contraceptive method from the start of mobilization through at least 6 months after Waskyra administration.Start of mobilization through minimum 6 months after administration

Coverage is not available when a medically eligible 10/10 HLA-identical matched stem cell donor is available for transplant. Requests may be reviewed case by case when an HLA-identical donor is identified but mobilized peripheral blood or bone marrow hematopoietic stem cell collection is not feasible.

Coverage is not available for patients who have received prior hematopoietic stem cell-based gene therapy.

Coverage is not available for patients with a history of hypersensitivity to dimethyl sulfoxide (DMSO), Waskyra’s active substance, or any of its excipients.

Coverage and Eligibility Parameters

Minimum patient age

Minimum patient age6 months or older

Eligible populationPediatric patients 6 months and older and adults

Age requirementThe patient must be at least 6 months of age for Waskyra coverage

Serum immunoglobulin G

Required serum IgG levelAbove 5 g/L

Testing requirementThe patient's immunoglobulin G serum level must be documented

Coverage conditionSerum IgG at or below 5 g/L does not meet the approval criterion

Prior Authorization and Provider Requirements

Prior Authorization

All Approval Criteria Must Be Met

Prior authorization is required. Approval requires documentation that all 16 approval criteria for Waskyra are met. Requests that do not demonstrate every applicable criterion will not be approved.

Submit clinical documentation supporting each of the 16 approval criteria before treatment, including the required diagnostic, eligibility, laboratory, monitoring, and safety information.

Documentation Required

Diagnosis and Transplant Eligibility

Document that the patient is at least 6 months old and has Wiskott-Aldrich Syndrome confirmed by molecular genetic testing with a severe-phenotype WAS mutation. Document transplant eligibility and donor evaluation, including that the patient does not have a medically eligible 10/10 HLA-identical matched stem cell donor; cases in which an identified donor cannot undergo feasible mobilized peripheral blood or bone marrow collection are reviewed case by case.

Document that the patient has not received an allogeneic HSCT within 6 months before screening, or, if received, has no residual donor cells and remains a candidate for autologous stem cell transplantation with adequate renal and hepatic function.

Therapy Background

Waskyra (etuvetidigene autotemcel) is indicated for pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome caused by a WAS gene mutation. The policy applies when hematopoietic stem cell transplantation is appropriate but no suitable HLA-matched related stem cell donor is available. Treatment involves stem-cell mobilization, conditioning-related procedures, infusion, and ongoing monitoring for serious complications.

Key Definitions

Wiskott-Aldrich Syndrome (WAS)

DefinitionA rare inherited disorder caused by a mutation in the WAS gene

Policy confirmationDiagnosis must be confirmed by molecular genetic testing

Required phenotypeThe patient must have a severe phenotype WAS mutation

Hematopoietic stem cell transplantation (HSCT)

DefinitionHematopoietic stem cell transplantation is the transplant option referenced by the policy

Eligibility requirementHSCT must be appropriate for the patient

Related donor condition

Initial Waskyra Therapy

Initial Therapy

Initial Waskyra therapy is covered only when all listed approval criteria are satisfied.

Initial Waskyra therapy

Clinical eligibility: Age at least 6 months; confirmed severe-phenotype WAS mutation; no medically eligible 10/10 HLA-identical donor; appropriate autologous transplant candidacy; and no qualifying prior HSCT or residual donor cells.All clinical eligibility requirements
Treatment and safety requirements: Single-agent use, no prior hematopoietic stem cell-based gene therapy, no relevant hypersensitivity, HIV-negative status, serum IgG above 5 g/L, required mobilization, monitoring, warnings review, pregnancy testing, and contraception must be documented or planned as applicable.All treatment and safety requirements

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicyWaskyra (etuvetidigene autotemcel) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation demonstrating that all 16 Waskyra approval criteria are satisfied.

SourceLink

On This Page

HLA donor match

Donor requirementNo medically eligible 10/10 HLA-identical matched stem cell donor for transplant

Donor relationshipThe indication applies when no suitable HLA-matched related stem cell donor is available

Case-by-case exceptionRequests may be reviewed case by case when an HLA-identical donor is identified but mobilized peripheral blood or bone marrow stem-cell collection is not feasible

Contraception duration

Contraception startAt the start of mobilization

Contraception durationThrough a minimum of 6 months after administration of Waskyra

Who must use contraceptionMales capable of fathering a child and females of childbearing age

Documentation Required

Required Laboratory and Safety Documentation

Provide the required laboratory, treatment-plan, monitoring, and reproductive-safety documentation. The patient must have a negative HIV test before mobilization and a serum IgG level above 5 g/L; females of reproductive potential require negative serum pregnancy testing before mobilization, again before conditioning, and again before Waskyra administration.

Document planned stem-cell mobilization with G-CSF and plerixafor according to established protocols.
Document monitoring for veno-occlusive disease, including liver function tests during the first month after infusion; monitoring for serious infection with prophylactic antimicrobials per local or institutional guidelines; and periodic post-treatment malignancy monitoring.
Document prescriber review of Waskyra warnings, precautions, and drug interactions, with appropriate patient-status monitoring.
Document effective contraception for males capable of fathering a child and females of childbearing age from the start of mobilization through at least 6 months after Waskyra administration.

Denial Risk

Failure to Meet Approval Criteria

Requests are denied when the approval criteria are not met. The provider must demonstrate compliance with all 16 criteria before coverage can be authorized.

Failure to provide sufficient documentation of any required criterion may result in denial.

Denial Risk

Clinical Eligibility or Safety Exclusion

Confirm that no clinical eligibility or safety exclusion applies before requesting authorization. Waskyra must be used as single-agent therapy, excluding lymphodepleting or conditioning therapy and pre-emptive anti-CD20 therapy while awaiting manufacture.

Do not request coverage when a medically eligible 10/10 HLA-identical matched stem cell donor is available, except for eligible case-by-case review when collection is not feasible.
The patient must not have received prior hematopoietic stem cell-based gene therapy.
The patient must not have a history of hypersensitivity to dimethyl sulfoxide (DMSO), Waskyra’s active substance, or any excipient.

No suitable HLA-matched related stem cell donor may be available

HLA-identical matched donor

Matching methodHuman leukocyte antigen (HLA) matching is used to evaluate donor compatibility

Required match assessmentThe policy evaluates whether a medically eligible 10/10 HLA-identical stem cell donor is available

Coverage relevanceWaskyra coverage requires that no medically eligible 10/10 HLA-identical matched donor be available for transplant, subject to the stated case-by-case exception