Medical Policy

Effective Date:01/01/2021 Title:Zolgensma (onasemnogene abeparvovec)
Revision Date: Document:BI669:00
CPT Code(s):J3399
Public Statement

Effective Date:

a)    This policy will apply to all services performed on or after the above revision date which will become the new effective date.

b)    For all services referred to in this policy that were performed before the revision date, contact customer service for the rules that would apply.

1)    Zolgensma requires prior authorization.

2)    Zolgensma is used to treat spinal muscular atrophy (SMA) in patients less than 2 years of age.

3)    Zolgensma is a specialty medication covered under the medical benefit.

Medical Statement

Initial Approval Criteria A. Spinal Muscular Atrophy (must meet all):

 

*Only for initial treatment dose; subsequent doses will not be covered.

1. Diagnosis of SMA Type I with onset of symptoms prior to 6 months of age;

2. Genetic testing confirming 1, 2, or 3 copies of SMN2 gene;

3. Genetic testing confirms the presence of one of the following (a, b, or c):

a. Homozygous deletions of SMN1 gene (e.g., absence of the SMN1 gene);

b. Homozygous mutation in the SMN1 gene (e.g., biallelic mutations of exon 7);

c. Compound heterozygous mutation in the SMN1 gene (e.g., deletion of SMN1 exon 7 (allele 1) and mutation of SMN1 (allele 2));

4. Prescribed by or in consultation with a neurologist;

5. Age < 2 years;

6. Documentation of one of the following baseline scores  (a or b):

a. Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorder (CHOP-INTEND) score;

b. Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestone score;

7. Documentation of both of the following (a and b):

a. Baseline laboratory tests demonstrating Anti-AAV9 antibody titers ≤ 1:50 as determined by ELISA binding immunoassay;

b. Baseline liver function test, platelet counts, and troponin-I;

8. Member does not have advanced SMA (e.g., complete paralysis of limbs, ventilator dependence for 16 or more hours per day, tracheostomy, or non-invasive ventilation beyond the use for sleep);

9. Member has not been previously treated with Zolgensma;

10. Zolgensma is not prescribed concurrently with Spinraza® or Evrysdi™;

11. If the member is currently on Spinraza, must meet the following (a and b):

a. Provider must submit evidence of clinical deterioration (e.g., sustained decrease in CHOP-INTEND score over a period of 3 to 6 months) upon completion of all loading doses of Spinraza;

b. Documentation of provider attestation of clinical deterioration and Spinraza discontinuation;

12. If the member is currently on Evrysdi, must meet the following (a and b):

a. Provider must submit evidence of clinical deterioration (e.g., sustained decrease in CHOP-INTEND score over a period of 3 to 6 months);

b. Documentation of provider attestation of clinical deterioration and Evrysdi discontinuation;

13. Member does not have an active viral infection;

14. Total dose does not exceed 1.1 x 1014 vector genomes (vg) per kilogram (kg).

 

Approval duration: 4 weeks (one time infusion per lifetime)

 

Continued Therapy

A. Spinal Muscular Atrophy

1. Re-authorization is not permitted.

Approval duration: Not applicable

 

Codes Used In This BI:

 

J3399 – injection, onasemnogene abeparvovec-xioi, per treatment, up to 5x10^15 vector genomes

Limits
Limited to one dose per lifetime.
Reference

1. Zolgensma Prescribing Information. Bannockburn, IL: AveXis, Inc.; May 2019. Available at: https://www.avexis.com/content/pdf/prescribing_information.pdf. Accessed August 20, 2020.

2. Mendell JR, Al-zaidy S, Shell R, et al. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. N Engl J Med. 2017;377(18):1713-1722.

3. ClinicalTrials.gov [Internet]. Identifier: NCT03421977, Long-Term Follow-up Study for Patients From AVXS-101-CL-101 (START). https://clinicaltrials.gov/ct2/show/NCT03421977. Accessed Februray 25, 2020.

4. ClinicalTrials.gov [Internet]. Identifier: NCT00381729, Study of Intrathecal Administration of AVXS-101 for Spinal Muscular Atrophy (STRONG). https://clinicaltrials.gov/ct2/show/NCT03381729. Accessed Februray 25, 2020.

5. ClinicalTrials.gov [Internet]. Identifier: NCT03505099, Pre-Symptomatic Study of Intravenous AVXS-101 in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT). https://clinicaltrials.gov/ct2/show/NCT03505099. Accessed Februray 25, 2020.

6. ClinicalTrials.gov [Internet]. Identifier: NCT03306277, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STR1VE). https://clinicaltrials.gov/ct2/show/NCT03306277. Accessed Februray 25, 2020.

7. ClinicalTrials.gov [Internet]. Identifier: NCT03461289, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STRIVE-EU). https://clinicaltrials.gov/ct2/show/NCT03461289. Accessed Februray 25, 2020.

8. Institute for Clinical and Economic Review (ICER): Final Evidence Report –Zolgensma and Spinraza. https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Final_Evidence_Report_052419.pdf. Accessed May 2019

9. 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. Neuromuscul Disord. 2018;28(2):103-115.

10. Finkel RS, Mercuri E, Meyer OH, et al. Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscul Disord. 2018;28(3):197-207.

11. Cobben JM, de Visser M, Scheffer H, et al. Confirmation of clinical diagnosis in requests for prenatal prediction of SMA type I. J Neurol Neurosurg Psychiatry 1993; 56: 319-21.

12. Maitre NL, Chorna O, Romeo DM, and Guzzetta A. Implementation of the Hammersmith Infant Neurological Examination in a High-Risk Infant Follow-Up Program. Pediatric Neurology 2016; 65:31-38.

13. Darras BT, Royden Jones H Jr, Ryan MM, et al. Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach. 2nd ed. London, UK: Elsevier; 2015.

14. Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology 2014; 83: 810-7.

15. De Sanctis R, Coratti G, Pasternak A, et al. Developmental milestones in type I spinal muscular atrophy. Neuromuscul Disord 2016; 26: 754-9.

Application to Products
This policy applies to all health plans administered by QualChoice, both those insured by QualChoice and those that are self-funded by the sponsoring employer, unless there is indication in this policy otherwise or a stated exclusion in your medical plan booklet. Consult the individual plan sponsor Summary Plan Description (SPD) for self-insured plans or the specific Evidence of Coverage (EOC) for those plans insured by QualChoice. In the event of a discrepancy between this policy and a self-insured customer’s SPD or the specific QualChoice EOC, the SPD or EOC, as applicable, will prevail. State and federal mandates will be followed as they apply.

Changes: QualChoice reserves the right to alter, amend, change or supplement benefit interpretations as needed.