MED.00129 Gene Therapy for Spinal Muscular Atrophy

This policy addresses onasemnogene abeparvovec-xioi gene replacement therapy for spinal muscular atrophy (SMA). It is covered as medically necessary only as a one-time infusion when the individual has confirmed 5q SMA due to bi-allelic SMN1 variants/deletions, is 2 years of age or younger at infusion, and either has no more than 3 SMN2 copies or had symptom onset before 6 months of age; otherwise—including repeat infusions or any other indications—it is considered investigational and not medically necessary.

"A one-time infusion of onasemnogene abeparvovec-xioi is consideredmedically necessaryin individuals with spinal muscular atrophy (SMA) whenallof the following criteria are met:"

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