Pharmacy Prior Authorization Criteria

Waskyra (etuvetidigene autotemcel) - PA, NF

For diagnosis of Wiskott-Aldrich Syndrome (WAS)
Indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cel transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cel donor is available

Prior Authorization, Non Formulary

Length of Approval: One Time Authorization Per Lifetime

Submission of medical records (e.g., chart notes) confirming diagnosis of Wiskott-Aldrich Syndrome (WAS)

AND

Submission of medical records (e.g., chart notes) confirming both of the following:

AND

Provider attests that mutation was detected by an FDA approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)

AND

Severe WAS mutation (e.g., mutations in exon 5-12, deletion mutation, insertion mutation, nonsense mutation) [A] [B]
Zhu clinical score of greater than or equal to 3

AND

Hematopoietic stem cell transplantation (HSCT) is appropriate for patient
No suitable human leukocyte antigen (HLA)-matched related stem cell donor is available

AND

AND

Patient has not received HSCT within 6 months prior to treatment with Waskyra
There is no evidence of residual HSCT donor cells
Patient has never received hematopoietic stem cell gene therapy treatment in their lifetime

AND

Prescribed by a physician with expertise in the treatment of Wiskott-aldrich syndrome at an authorized treatment center for Waskyra with shared decision-making regarding treatment risks

1970-01-01, 2026-10-09

Waskyra Prescribing information. Fondazione Telethon ETS, Rome, Italy. July 2026.
Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS) ClinicalTrials.gov. NCT01515462
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome. ClinicalTrials.gov. NCT3837483
Wiscott Aldrich syndrome, Hans D Ochs, MD. UpToDate, Aug 2026.
The WAS gene and it's mutations. available at ww.wiscott.org. Accessed September 2026.

Both clinical trials evaluating the use of Waskyra in WAS only selected patients with a WAS mutation predictive of severe disease rather than phenotypes associated with milder disease (2,3)
Mutations have been found on all the 12 exons in WAS patients. Usually, the mutations in the first 4 exons are associated with milder disease whereas the ones from exon 5-12 cause more severe disease. Most patients with milder disease were noted to have missense mutations or occasionally splice site mutations. The majority of patient with severe WAS had deletions, insertions, nonsense mutations or splice mutations. (5)