Waskyra (etuvetidigene autotemcel) coverage for Wiskott-Aldrich Syndrome
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Defines prior authorization, dosing, eligibility, and administration requirements for Waskyra (etuvetidigene autotemcel) provided to members with Wiskott-Aldrich Syndrome under Viva Health.
No material clinical or coverage changes in this revision.
Coverage Criteria
Initial Therapy (Medical Necessity)
Covered when ALL of the following are met for Wiskott-Aldrich Syndrome (WAS):
AND member meets one of
- Severe phenotype: Severe-phenotype WAS mutation
- Clinical severity: Signs and symptoms consistent with classic severe WAS phenotype (Zhu-Clinical or WAS-Severity Score >= 3)
- Absent protein: Absent WAS protein (WASP) expression
See dosing/administration for collection and back-up requirements
Renewal Therapy
Renewal criteria
No renewals permitted per policy
Coverage is excluded for members with any FDA-labeled contraindication to Waskyra. Examples include: hypersensitivity to etuvetidigene autotemcel or any excipients, prior allogeneic hematopoietic stem cell transplant (HSCT) within 6 months before screening or HSCT with evidence of residual donor cells, previous hematopoietic stem cell gene therapy, or other conditions that contraindicate the required mobilization and conditioning regimens. These contraindications must be assessed and documented prior to approval.
Prior authorization is granted for a single treatment only. The policy provides initial authorization for one dose total and explicitly states that prior authorization may not be renewed. Requests for additional or repeat doses beyond that single authorized treatment are not permitted.
Initial Therapy (Drug-specific)
Initial Therapy (Drug-specific)
Initial approval requires demonstration of clinical and genetic criteria plus adherence to preparatory, administration, and monitoring requirements.
Do not begin conditioning until complete infusion bag(s) and back-up collection are confirmed available
Only one bag should be infused at a time when multiple bags are required
Continuation / Renewal
Continuation Therapy
Continuation/renewal not permitted
Coding and Quantity Limits
| J3386 | Injection, etuvetidigene autotemcel, per treatment; 1 billable unit = 1 treatment |
| D82.0 | Wiskott-Aldrich syndrome |
Provider Actions & Documentation
Prior authorization — single dose
Prior authorization is required and will be provided for one dose only. One treatment equals one billable unit (J3386); prior authorization may NOT be renewed.
- HCPCS code for billing per treatment: J3386
Dosing and preparatory steps — mobilization, collection, premedication, and volume/DMSO limits
Follow required dosing, mobilization, collection, storage, and administration steps prior to conditioning and infusion: perform mobilization and apheresis to collect CD34+ cells, collect and cryopreserve a back-up supply of ≥ 3 x 10^6 CD34+ cells/kg, do not begin reduced‑intensity conditioning until complete infusion bag(s) are received and back‑up confirmed, premedicate (e.g., IV chlorpheniramine 0.2 mg/kg, max 10 mg, 15–30 minutes before infusion), and ensure product volume and DMSO limits are observed when multiple bags are used.
- Collect required CD34+ cells by apheresis after mobilization with G‑CSF and plerixafor.
- Store back-up collection (≥ 3 x 10^6 CD34+ cells/kg) before initiating conditioning.
- Do not begin conditioning until complete set of infusion bag(s) is received and back‑up confirmed.
- Premedicate with IV chlorpheniramine (0.2 mg/kg, max 10 mg) or equivalent 15–30 minutes prior to infusion.
- When multiple bags are needed, ensure total DMSO < 1% of estimated plasma volume and total product volume < 20% of estimated plasma volume; infuse one bag at a time.
Required supporting documentation — submit with PA request
Submit supporting clinical documentation with the PA request via direct upload to the PA web portal or fax. Required records include medical records, chart notes, laboratory results, and confirmatory diagnostics (e.g., genetic/mutational testing) to document diagnosis, step therapy, and clinical markers.
- Include documentation of WAS diagnosis (WAS gene mutation testing) and clinical severity or WASP expression when applicable.
- Provide records of mobilization/apheresis and back‑up collection details.
- Provide lab results and any other confirmatory diagnostics referenced in the request.
Documentation submission required — risk of denial or delay if not provided
Failure to submit the required supporting clinical documentation (medical records, chart notes, lab results, confirmatory diagnostics) at the time of request may result in denial of the authorization due to inability to establish medical necessity or may delay processing.
- Submit records via PA web portal upload or by fax as instructed.
- Missing documentation will be reviewed at time of submission and can lead to denial.
Definitions
Step Therapy Requirements
| Step | Requirement | Timing / Notes |
|---|---|---|
| {"text":"1","status":""}|{"text":"Perform mobilization with G-CSF and plerixafor to mobilize CD34+ hematopoietic stem cells for collection.","status":""}|{"text":"Before apheresis; mobilization precedes collection and conditioning (per policy dosing/administration).","status":""} | ||
| {"text":"2","status":""}|{"text":"Perform apheresis to collect the CD34+ cells required for Waskyra manufacturing.","status":""}|{"text":"Collect cells after mobilization and prior to reduced-intensity conditioning; collected product used for manufacturing.","status":""} | ||
| {"text":"3","status":""}|{"text":"Collect and cryopreserve a back-up supply of CD34+ stem cells containing at least 3 x 10^6 CD34+ cells/kg.","status":""}|{"text":"Must be completed before initiating reduced-intensity conditioning and infusion; backup stored for potential rescue (product compromise, primary engraftment failure, prolonged aplasia).","status":""} | ||
| {"text":"4","status":""}|{"text":"Confirm receipt and on-site storage of the complete set of infusion bag(s) and availability of the back-up collection prior to beginning conditioning.","status":""}|{"text":"Do not begin conditioning until complete infusion bag(s) received and back-up confirmed at administration site.","status":""} | ||
| {"text":"5","status":""}|{"text":"Ensure product volume and DMSO limits are observed when multiple bags are used: total DMSO administered < 1% of estimated plasma volume; maximum product volume < 20% of estimated plasma volume.","status":""}|{"text":"When more than one bag is needed, only one bag should be infused at a time; limits apply to total DMSO and product volume.","status":""} |
Site of Care Requirements
Hospital outpatient — on‑site storage and back‑up collection required
For hospital outpatient administration, ensure on‑site receipt and storage of the complete infusion bag(s) and confirmation of the back‑up collection prior to initiating conditioning or infusion; observe product volume and DMSO limits when multiple bags are required.
- Do not begin conditioning until the complete set of infusion bag(s) has been received and stored at the administration site and back‑up collection availability is confirmed
- When more than one bag is needed, ensure total DMSO administered remains < 1% of the member's estimated plasma volume and maximum product volume remains < 20% of estimated plasma volume
Background
Wiskott-Aldrich Syndrome (WAS) is an X-linked primary immunodeficiency caused by mutations in the WAS gene that result in impaired immune function. Waskyra (etuvetidigene autotemcel) is an autologous, lentiviral vector–modified CD34+ hematopoietic stem cell gene therapy intended to restore WAS protein expression and improve immune function. Treatment requires mobilization with G-CSF and plerixafor, apheresis with collection and cryopreservation of back-up CD34+ cells, reduced‑intensity conditioning prior to infusion, and close post-infusion monitoring for infection, maintenance of immunoglobulin G > 5 g/L, liver function to assess for veno‑occlusive disease, and surveillance for lentiviral vector–mediated insertional oncogenesis and secondary malignancies.
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