Anthem Blue Cross Connecticut Commercial Clinical Policy

Anthem Blue Cross Connecticut MED.00129 Gene Therapy for Spinal Muscular Atrophy Form


Gene Therapy for Spinal Muscular Atrophy (Onasemnogene abeparvovec-xioi)

Indications

(573740) Has the patient been diagnosed with spinal muscular atrophy (SMA) as documented by a bi-allelic SMN1 5q gene variant or deletions? 
(573741) Does the patient have no more than 3 copies of SMN2, or onset of SMA-associated signs and symptoms before 6 months of age? 
(573742) Is the patient two years of age or younger at the time of vector infusion? 
(573743) Is the patient's Anti-adeno-associated viral vector, serotype 9 (AAV9) antibody titer less than or equal to 1:50? 
(573744) Does the patient not use invasive ventilatory support (tracheotomy with positive pressure) or non-invasive ventilator support (BiPAP) for more than 16 hours per day as a result of advanced SMA disease? 
(573745) Does the patient not have any serious concomitant illness (for example, severe liver or kidney disease, symptomatic cardiomyopathy, active viral infection)? 

Effective Date

NA

Last Reviewed

11/09/2023

Original Document

  Reference



This document addresses gene therapy for spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. One gene therapy product, Zolgensma® (onasemnogene abeparvovec-xioi), has been approved by the Food and Drug Administration (FDA).

Note: Please refer to clinical pharmacy criteria for information regarding other disease-modifying treatments for SMA; for example: nusinersen (Spinraza) or risdiplam (Evyrsdi).

Position Statement

Medically Necessary:

A one-time infusion of onasemnogene abeparvovec-xioi is considered medically necessary in individuals with spinal muscular atrophy (SMA) when all of the following criteria are met:

  1. Confirmed SMA diagnosis as documented by a bi-allelic SMN1 5q gene variant or deletions and either of the following:
    1. No more than 3 copies of SMN2; or
    2. Onset of SMA-associated signs and symptoms before 6 months of age; and
  2. Two years of age or younger at the time of vector infusion; and
  3. Anti-adeno-associated viral vector, serotype 9 (AAV9) antibody titer less than or equal to 1:50; and
  4. No use of invasive ventilatory support (tracheotomy with positive pressure) or use of non-invasive ventilator support (BiPAP) for more than 16 hours per day as a result of advanced SMA disease; and
  5. No serious concomitant illness (for example, severe liver or kidney disease, symptomatic cardiomyopathy, active viral infection).

Investigational and Not Medically Necessary:

Onasemnogene abeparvovec-xioi is considered investigational and not medically necessary when the criteria above are not met, including for repeat infusions, and for all other indications.