Pharmacy Prior Authorization Criteria

Zolgensma (onasemnogene abeparvovec-xioi), Itvisma (onasemnogene abeparvovec-brve) - 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.

Itvisma (onasemnogene abeparvovec-brve)

• For diagnosis of Spinal Muscular Atrophy (SMA)
  Indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in survival motor neuron 1 (SMN1) gene.

Criteria

Zolgensma

Prior Authorization, Non-Formulary

Length of Approval: 1 Time Authorization in Lifetime

• Submission of medical records (e.g., chart notes) confirming the mutation or deletion of genes in chromosome 5q as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA) 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 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 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 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 [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) confirming anti-AAV9 antibody titers are less than or equal to 1:50 [1]
AND
• Both of the following:
• Provider attests that the patient has never received onasemnogene abeparvovec (e.g., Zolgensma, Itvisma) treatment in their lifetime
AND
• Provider attests that the patient is not receiving concomitant chronic survival 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

Itvisma

Prior Authorization, Non-Formulary

Length of Approval: 1 Time Authorization in Lifetime

• Both of the following:
• Submission of medical records (e.g., chart notes) confirming diagnosis of spinal muscular atrophy (SMA)
AND
• Submission of medical records (e.g., chart notes) confirming the presence of a SMN1 gene mutation as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
AND
• Patient is 2 years of age or older
AND
• Both of the following:
• Provider attests that patient has not received treatment with onasemnogene abeparvovec (e.g., Zolgensma, Itvisma) in their lifetime [F]
AND
• Provider attests that patient is not receiving concomitant chronic survival motor neuron (SMN) modifying therapy for the treatment of SMA (e.g., Spinraza, Evrysdi) [E]
AND
• Patient will commence systemic corticosteroids equivalent to oral prednisolone at 1mg per kg of body weight per day (mg/kg/day), starting one day prior to Itvisma therapy and planned for a total of 30 days
AND
• Submission of medical records (e.g., chart notes) confirming the absence of hepatic dysfunction [e.g., alanine aminotransferase (ALT), total bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH) less than or equal to the upper limit of normal (ULN)]
AND
• Provider attests that patient has not had clinical signs or symptoms of infection within 30 days prior to treatment
AND
• Both of the following [G]:
• Patient can sit independently
AND
• Patient has never had the ability to walk independently
AND
• Submission of medical records (e.g., chart notes) confirming anti-AAV9 antibody titers are less than or equal to 1:50 [G]
AND
• Patient’s clinical signs and symptoms onset occurred at greater than or equal to 6 months of age [G]
AND
• Patient is not dependent on any of the following [G]:
• Invasive ventilation
• Awake noninvasive ventilation for greater than 6 hours during a 24-hour period
• Noninvasive ventilation for greater than 12 hours during a 24-hour period
AND
• Both of the following [G]:
• Patient has not received surgery for scoliosis or hip fixation in the 12 months prior to approval
AND
• Patient has no planned surgery for scoliosis or hip fixation within the next 64 weeks following therapy
AND
• Prescribed by a neurologist with expertise in the diagnosis and treatment of SMA

P & T Revisions

2026-01-23, 2025-05-13, 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. February 2025.
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.
11. Itvisma Prescribing Information. Novartis Gene Therapies, Inc. Bannockburn, IL November 2025.

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End Notes

1. This is the definition that the clinical trials used. Also consistent with clinical guidelines. [2-8]
2. There were 3 key clinical trials for Zolgensma (START, STR1VE, SPR1NT). START and STR1VE only enrolled patients with SMA Type 1 and SPR1NT enrolled pre-symptomatic SMA patients. [2-5]
3. Exclusion criteria found in clinical trials. [2-5]
4. A recent European ad-hoc consensus statement on SMA stated that there currently is no published evidence that the combination of two disease modifying therapies (e.g., Spinraza and Zolgensma) is superior to any single treatment alone. RESPOND is a phase 4 trial that will assess the efficacy and safety of Spinraza in patients with suboptimal clinical response to Zolgensma. It is planned to begin enrollment in 2021. [9-10]
5. Spinraza and Evrysdi are listed as examples of SMN-targeted therapies in the exclusion criteria of the pivotal trial. Study 2, as per Itvisma Prescribing information, is an open-label single arm study evaluating the safety of Itvisma in patients with SMA who were previously treated with nusinersen or risdiplam. No additional safety concerns are noted.
6. As per Itvisma Prescribing information, patients previously treated with Zolgensma should not be treated with Itvisma. [11]
7. Adapted from key inclusion and exclusion criteria found in the pivotal trial.

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Revision History

• 2026-01-23: Addition of New Formulation. Itvisma PA added. 2026 Annual Review. Combined PA and NF criteria into one criteria bucket as criteria aligns. Update to standard genetic testing verbiage. Removed reference to tracheostomy. Replaced "documenting" verbiage with "confirming" to align with standard verbiage throughout. Updated age criterion to align with label.
• 2025-05-13: 2025 Annual Review. Background updates only.
• 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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