Asset Publisher
Zolgensma® (onasemnogene abeparvovec-xioi)
Policy Number: PH-468
(Intravenous)
Last Review Date: 08/08/2023
Date of Origin: 06/01/2019
Dates Reviewed: 06/2019, 08/2020, 08/2021, 08/2022, 08/2023
- Length of Authorization
Coverage will be provided for one dose and may not be renewed.
- Dosing Limits
- Quantity Limit (max daily dose) [NDC Unit]:
- 1 kit (based on weight chart below)
- Max Units (per dose and over time) [HCPCS Unit]:
- 1 kit (based on weight chart below)
- Initial Approval Criteria
Submission of medical records (chart notes) related to the medical necessity criteria is REQUIRED on all requests for authorizations. Records will be reviewed at the time of submission. Please provide documentation related to diagnosis, step therapy, and clinical markers (i.e. genetic and mutational testing) supporting initiation when applicable. Please provide documentation via direct upload through the PA web portal or by fax. |
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 SMA phenotype 1 confirmed by one or more of the following:
- Patient must have 1-2 copies of the SMN2 gene; OR
- Patient has 3 copies of the SMN2 gene in the absence of the c.859G>C single base substitution modification in exon 7; AND
- Patient must have a baseline anti-AAV9 antibody titer of ≤ 1:50 measured by ELISA; 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
- Will not be used in combination with other agents for SMA (e.g., nusinersen, risdiplam, etc.)
† FDA Approved Indication(s); ‡ Compendium Recommended Indication(s); Ф Orphan Drug
- Renewal Criteria 1
Coverage cannot be renewed.
- Dosage/Administration 1
Indication |
Dose |
SMA1 |
Preparing for Administration:
Zolgensma Infusion:
|
NOTE:
|
- 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:
Zolgensma kit sizes:
Patient Weight (kg) |
NDC |
Patient Weight (kg) |
NDC |
2.6 – 3.0 |
71894-0120 |
12.1 – 12.5 |
71894-0139 |
3.1 – 3.5 |
71894-0121 |
12.6 – 13.0 |
71894-0140 |
3.6 – 4.0 |
71894-0122 |
13.1 – 13.5 |
71894-0141 |
4.1 – 4.5 |
71894-0123 |
13.6 – 14.0 |
71894-0142 |
4.6 – 5.0 |
71894-0124 |
14.1 – 14.5 |
71894-0143 |
5.1 – 5.5 |
71894-0125 |
14.6 – 15.0 |
71894-0144 |
5.6 – 6.0 |
71894-0126 |
15.1 – 15.5 |
71894-0145 |
6.1 – 6.5 |
71894-0127 |
15.6 – 16.0 |
71894-0146 |
6.6 – 7.0 |
71894-0128 |
16.1 – 16.5 |
71894-0147 |
7.1 – 7.5 |
71894-0129 |
16.6 – 17.0 |
71894-0148 |
7.6 – 8.0 |
71894-0130 |
17.1 – 17.5 |
71894-0149 |
8.1 – 8.5 |
71894-0131 |
17.6 – 18.0 |
71894-0150 |
8.6 – 9.0 |
71894-0132 |
18.1 – 18.5 |
71894-0151 |
9.1 – 9.5 |
71894-0133 |
18.6 – 19.0 |
71894-0152 |
9.6 – 10.0 |
71894-0134 |
19.1 – 19.5 |
71894-0153 |
10.1 – 10.5 |
71894-0135 |
19.6 – 20.0 |
71894-0154 |
10.6 – 11.0 |
71894-0136 |
20.1 – 20.5 |
71894-0155 |
11.1 – 11.5 |
71894-0137 |
20.6 – 21.0 |
71894-0156 |
11.6 – 12.0 |
71894-0138 |
|
|
- References
- Zolgensma [package insert]. Bannockburn, IL; AveXis, Inc., February 2023. Accessed July 2023.
- 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.
- 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.
- Prior TW, Finanger E. Spinal muscular atrophy. GeneReviews. www.ncbi.nlm.nih.gov/books/NBK1352/. Initial Posting: February 24, 2000; Last Revision: December 3, 2022. Accessed on July 10, 2023.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- (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
ICD-10 |
ICD-10 Description |
G12.0 |
Infantile spinal muscular atrophy, type I [Werdnig-Hoffmann] |
Appendix 2 – Centers for Medicare and Medicaid Services (CMS)
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 Determination (NCD), Local Coverage Articles (LCAs) and Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. They can be found at: http://www.cms.gov/medicare-coverage-database/ search.aspx. Additional indications may be covered at the discretion of the health plan.
Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCA/LCD): N/A
Medicare Part B Administrative Contractor (MAC) Jurisdictions |
||
Jurisdiction |
Applicable State/US Territory |
Contractor |
E (1) |
CA, HI, NV, AS, GU, CNMI |
Noridian Healthcare Solutions, LLC |
F (2 & 3) |
AK, WA, OR, ID, ND, SD, MT, WY, UT, AZ |
Noridian Healthcare Solutions, LLC |
5 |
KS, NE, IA, MO |
Wisconsin Physicians Service Insurance Corp (WPS) |
6 |
MN, WI, IL |
National Government Services, Inc. (NGS) |
H (4 & 7) |
LA, AR, MS, TX, OK, CO, NM |
Novitas Solutions, Inc. |
8 |
MI, IN |
Wisconsin Physicians Service Insurance Corp (WPS) |
N (9) |
FL, PR, VI |
First Coast Service Options, Inc. |
J (10) |
TN, GA, AL |
Palmetto GBA, LLC |
M (11) |
NC, SC, WV, VA (excluding below) |
Palmetto GBA, LLC |
L (12) |
DE, MD, PA, NJ, DC (includes Arlington & Fairfax counties and the city of Alexandria in VA) |
Novitas Solutions, Inc. |
K (13 & 14) |
NY, CT, MA, RI, VT, ME, NH |
National Government Services, Inc. (NGS) |
15 |
KY, OH |
CGS Administrators, LLC |
ZOLGENSMA® (onasemnogene abeparvovec-xioi) Prior Auth Criteria |
|