Pharmacy Prior Authorization Criteria

Zolgensma (onasemnogene abeparvovec-xioi) - PA, NF

Indications for Prior Authorization

Zolgensma (onasemnogene abeparvovec-xioi)

• For diagnosis of Spinal Muscular Atrophy (SMA)
  Indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

  Limitation of Use: • The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated. • The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated.

Criteria

Zolgensma

Prior Authorization

Length of Approval: 1 Time Authorization in Lifetime

• The mutation or deletion of genes in chromosome 5q resulting in one of the following: [1-8, A]
• Homozygous gene deletion or mutation of SMN1 gene (e.g., homozygous deletion of exon 7 at locus 5q13)
OR
• Compound heterozygous mutation of SMN1 gene (e.g., deletion of Survival of Motor Neuron 1 [SMN1] exon 7 [allele 1] and mutation of SMN1 [allele 2])
AND
• One of the following:
• Both of the following: [1-5]
• Diagnosis of symptomatic spinal muscular atrophy (SMA) confirmed by a neurologist with expertise in the diagnosis and treatment of SMA [B]
AND
• Patient is less than or equal to 2 years of age
OR
• All of the following:
• Diagnosis of SMA based on the results of SMA newborn screening
AND
• Patient has 4 copies or less of Survival of Motor Neuron 2 (SMN 2)
AND
• Patient is less than or equal to 6 months of age [2-5]
AND
• Patient is not dependent on invasive ventilation or tracheostomy [2-5, C]
AND
• Patient is not dependent on the use of non-invasive ventilation beyond use for naps and nighttime sleep [2-5, C]
AND
• Documentation of anti-AAV9 antibody titers being less than or equal to 1:50 [1]
AND
• Patient is not to receive concomitant chronic survivor motor neuron (SMN) modifying therapy for the treatment of SMA (e.g. Spinraza, Evrysdi) [2-5,D]
AND
• Prescribed by a neurologist with expertise in the diagnosis and treatment of SMA
AND
• Patient has never received Zolgensma treatment in their lifetime [1]

Zolgensma

Non Formulary

Length of Approval: 1 Time Authorization in Lifetime

• Submission of medical records (e.g., chart notes) documenting the mutation or deletion of genes in chromosome 5q resulting in one of the following: [1-8, A]
• Homozygous gene deletion or mutation of SMN1 gene (e.g., homozygous deletion of exon 7 at locus 5q13)
OR
• Compound heterozygous mutation of SMN1 gene (e.g., deletion of Survival of Motor Neuron 1 [SMN1] exon 7 [allele 1] and mutation of SMN1 [allele 2])
AND
• One of the following:
• Both of the following: [1-5]
• Diagnosis of symptomatic spinal muscular atrophy (SMA) confirmed by a neurologist with expertise in the diagnosis and treatment of SMA [B]
AND
• Patient is less than or equal to 2 years of age
OR
• All of the following:
• Diagnosis of SMA based on the results of SMA newborn screening
AND
• Patient has 4 copies or less of Survival of Motor Neuron 2 (SMN 2)
AND
• Patient is less than or equal to 6 months of age [2-5]
AND
• Patient is not dependent on invasive ventilation or tracheostomy [2-5, C]
AND
• Patient is not dependent on the use of non-invasive ventilation beyond use for naps and nighttime sleep [2-5, C]
AND
• Submission of medical records (e.g., chart notes) documenting anti-AAV9 antibody titers being less than or equal to 1:50 [1]
AND
• Patient is not to receive concomitant chronic survivor motor neuron (SMN) modifying therapy for the treatment of SMA (e.g. Spinraza, Evrysdi) [2-5,D]
AND
• Prescribed by a neurologist with expertise in the diagnosis and treatment of SMA
AND
• Patient has never received Zolgensma treatment in their lifetime [1]

P & T Revisions

2024-06-05, 2023-06-19, 2022-11-29, 2022-06-16, 2021-06-07, 2021-04-20, 2020-11-20, 2020-06-19

References

1. Zolgensma Prescribing Information. AveXis Inc. Bannockburn, IL. October 2023.
2. Mendell J.R., Al-Zaidy S, Shell R, etc. Single-Dose Gene Replacement Therapy for Spinal Muscular Atrophy. New Eng J of Med. 2017; 377:1713-22.
3. 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;54(2):179-185.
4. Day JW, Chiriboga CA, Crawford TO, et al. AVXS-101 gene-replacement therapy for spinal muscular atrophy type 1: phase 3 study (STR1VE) update. Poster presented at: The 71st Annual American Academy of Neurology Meeting, Philadelphia PA, May 4-10, 2019.
5. Strauss KA, Swoboda KJ, Farrar MA, et al. AVXS-101 gene-replacement therapy in presymptomatic spinal muscular atrophy: SPR1NT study update. Poster presented at the 71st Annual American Academy of Neurology Meeting; May 4-10; 2019; Philadelphia, PA.
6. Markowitz JA, Sing P, Darras BT. Spinal muscular atrophy: a clinical and research update. Pediatr Neurol. 2012;46(1):1-12.
7. Wang CH, Finkel RS, Bertini ES, et al. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007;22(8):1027-1049.
8. Mercuri E, Finkel RS, Muntoni F, et al. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. J Neuromuscul Dis. 2018;28(2):103-115.
9. Kirschner J, Butoianu N, Goemans N, et al. European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy. Eur J Paediatr Neurol. 2020. https://doi.org/10.1016/j.ejpn.2020.07.001.
10. Biogen. Biogen plans to initiate phase 4 study evaluating benefit of Spinraza® (nusinersen) in patients treated with Zolgensma® (onasemnogene abeparvovec). https://investors.biogen.com/news-releases/news-release-details/biogen-plans-initiate-phase-4-study-evaluating-benefit-spinrazar. July 21, 2020. Accessed October 6, 2020.

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Revision History

• 2024-06-05: 2024 Annual Review. No criteria changes. Updated references.
• 2023-06-19: Annual Review
• 2022-11-29: update guideline
• 2022-06-16: Annual review - removed submission of medical records requirement from prior authorization criteria. Updated references.
• 2021-06-07: Annual Review
• 2021-04-20: Updated guideline
• 2020-11-20: Annual Review Updates
• 2020-06-19: Annual review

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