Last Review Date: 08/01/2024

Date of Origin: 06/01/2019

Dates Reviewed: 06/2019, 08/2020, 08/2021, 08/2022, 08/2023, 08/2024

1. Length of Authorization

Coverage will be provided for one dose and may not be renewed.

2. Dosing Limits

1. Quantity Limit (max daily dose) [NDC Unit]:

• 1 kit (based on weight chart below)

2. Max Units (per dose and over time) [HCPCS Unit]:

• 1 billable unit (1 treatment of up to 5x10¹⁵ vector genomes)

3. Initial Approval Criteria

Coverage is provided in the following conditions:

Spinal Muscular Atrophy (SMA) † Ф ^1-11

• Patient must be less than 2 years of age; AND
• Patient has a diagnosis of 5q spinal muscular atrophy confirmed by either bi-allelic deletion or dysfunctional point mutation of the SMN1 gene; AND
• Patient must have 4 or fewer copies of the SMN 2 gene; AND
• Baseline liver function will be assessed prior to initiating therapy and will continue to be monitored for at least 3 months after therapy; AND
• Used concomitantly with systemic corticosteroids (see dosage/administration below); AND
• Patient does not have advanced disease (complete limb paralysis, permanent ventilation support, etc.); AND
• Patient must not have previously been administered onasemnogene abeparvovec-xioi; AND
• Will not be used in combination with other agents for SMA (e.g., nusinersen, risdiplam, etc.)

† FDA Approved Indication(s); ‡ Compendia Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ¹

Coverage cannot be renewed.

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS code:

• J3399 – Injection, onasemnogene abeparvovec-xioi, per treatment, up to 5x10¹⁵ vector genomes; 1 billable unit = 1 treatment, up to 5x10¹⁵ vector genomes

NDC(s):

Zolgensma kits:

7. References

1. Zolgensma [package insert]. Bannockburn, IL; Novartis Gene Therapies, Inc., October 2023. Accessed June 2024.
2. Mendell JR, Al-Zaidy S, Shell R. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377(18):1713-1722. doi: 10.1056/NEJMoa1706198.
3. Wang CH, Finkel RS, Bertini ES, et al. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007 Aug;22(8):1027-49.
4. Prior TW, Leach, ME, Finanger E. Spinal muscular atrophy. GeneReviews. www.ncbi.nlm.nih.gov/books/NBK1352/. Initial Posting: February 24, 2000; Last Revision: December 3, 2020. Accessed on June 18, 2024.
5. Dabbous O, Maru B, Jansen JP, et al. Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther. 2019 May;36(5):1164-1176.
6. Al-Zaidy S, Pickard AS, Kotha K, et al. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb;54(2):179-185.
7. Al-Zaidy SA, Kolb SJ, Lowes L, et al. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019;6(3):307-317. doi: 10.3233/JND-190403.
8. Lowes LP, Alfano LN, Arnold WD, et al. Impact of age and motor function in a phase 1/2A study of infants with SMA type 1 receiving single-dose gene replacement therapy. Pediatr Neurol. 2019;98:39-45.
9. Day JW, Finkel RS, Chiriboga CA, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17.
10. Mendell JR, Al-Zaidy SA, Lehman KJ, et al. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 May 17;e211272. doi: 10.1001/jamaneurol.2021.1272.
11. (ICER) IfCaER. Spinraza and Zolgensma for Spinal Muscular Atrophy: Effectiveness and Value. Final Evidence Report. April 3, 2019 (Updated May 24, 2019) 2019.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCA/LCD): N/A