Nusinersen (Spinraza™)
IMPORTANT REMINDER
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.
POLICY
Nusinersen for the treatment of spinal muscular atrophy (SMA) is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Nusinersen for the treatment of other conditions/diseases is considered investigational.
MEDICAL APPROPRIATENESS
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.
Universal Criteria
Patient must not have previously received treatment with SMA gene therapy (i.e., onasemnogene abeparvovec-xioi); AND
Patient will not use in combination with other agents for SMA (e.g., onasemnogene abeparvovec, risdiplam, etc.); AND
Patient must not have advanced disease (complete limb paralysis, permanent ventilation support, etc.); AND
Patient must have the following laboratory tests at baseline and prior to each administration*: platelet count, prothrombin time; activated partial thromboplastin time, and quantitative spot urine protein testing; AND
Spinal Muscular Atrophy (SMA)
Patient retains meaningful voluntary motor function (e.g., manipulate objects using upper extremities, ambulate, etc.); AND
Patient must have a diagnosis of 5q spinal muscular atrophy confirmed by either homozygous deletion of the SMN1 gene or dysfunctional mutation of the SMN1 gene; AND
Patient must have a diagnosis of SMA phenotype I, II or III; AND
Patient has ≤ 3 copies of the SMN2 gene (Note: Patients with >3 copies of the SMN2 gene will be reviewed on a case-by-case basis); OR
Patient has symptomatic disease (i.e., impaired motor function and/or delayed motor milestones); AND
Baseline documentation of one or more of the following:
Motor function/milestones, including but not limited to, the following validated scales: Hammersmith Infant Neurologic Exam (HINE), Hammersmith Functional Motor Scale Expanded (HFMSE), Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), Bayley Scales of Infant and Toddler development Third Ed. (BSID-III), 6-minute walk test (6MWT), upper limb module (ULM), motor function measure 32 (MFM32), revised upper limb module (RULM), etc.
Respiratory function tests [e.g., forced vital capacity (FVC), etc.]
Exacerbations necessitating hospitalization and/or antibiotic therapy for respiratory infection in the preceding year/timeframe
Patient weight (for patients without a gastrostomy tube)
*Laboratory tests should be obtained within several days prior to administration
RENEWAL CRITERIA
Patient continues to meet the universal and other indication specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in Initial Approval Criteria; AND
Absence of unacceptable toxicity which would preclude safe administration of the drug. Examples of unacceptable toxicity include significant renal toxicity, thrombocytopenia, coagulation abnormalities, etc.; AND
Patient has responded to therapy compared to pretreatment baseline in one or more of the following:
Stability or improvement in net motor function/milestones, including but not limited to, the following: validated scales: Hammersmith Infant Neurologic Exam (HINE), Hammersmith Functional Motor Scale Expanded (HFMSE), Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), ), Bayley Scales of Infant and Toddler development Third Ed. (BSID-III), 6-minute walk test (6MWT), upper limb module (ULM), motor function measure 32 (MFM32), revised upper limb module (RULM), etc.
Stability or improvement in respiratory function tests [e.g., (FVC), etc.]
Reduction in exacerbations necessitating hospitalization and/or antibiotic therapy for respiratory infection in the preceding year timeframe
Stable or increased patient weight (for patients without a gastrostomy tube)
Slowed rate of decline in the aforementioned measures
LENGTH OF AUTHORIZATION
Coverage will be provided annually and may be renewed
APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
ADDITIONAL INFORMATION
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
REFERENCES
1. Spinraza [package insert]. Cambridge, MA; Biogen, Inc.; June 2020. Accessed July 2022.
2. 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.
3. 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 12, 2023.
4. Finkel RS, Mercuri E, Darras BT, et al; for the ENDEAR Study Group. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723- 1732.Finkel RS, Mercuri E, Darras BT, et al; for the ENDEAR Study Group. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723-1732.
5. Mercuri E, Darras BT, Chiriboga CA, et al; for the CHERISH Study Group. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.
6. 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.
7. Kichula E, Duong T, Glanzman A, et al. Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Feasibility for Individuals with Severe Spinal Muscular Atrophy II (S46.004). Neurology Apr 2018, 90 (15 Supplement) S46.004
8. De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019 Nov;29(11):842-856. doi: 10.1016/j.nmd.2019.09.007. Epub 2019 Sep 12.
9. Michelson D, Ciafaloni E, Ashwal S, et al. Evidence in focus: Nusinersen use in spinal muscular atrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology. Neurology. 2018 Nov 13;91(20):923-933. doi: 10.1212/WNL.0000000000006502. Epub 2018 Oct 12.
10. Darras BT, Chiriboga CA, Iannaccone ST, et al. Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies. Neurology. 2019;92(21):e2492-e2506
11. Finikel 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 Mar;28(3):197-207. doi: 10.1016/j.nmd.2017.11.004. Epub 2017 Nov 23.
12. (ICER) IfCaER . Spinraza and Zolgensma for Spinal Muscular Atrophy: Effectiveness and Value. Final Evidence Report. April 3, 2019 (Updated May 24, 2019) 2019.
13. Kichula E, Duong T, Glanzman A, et al. Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Feasibility for Individuals with Severe Spinal Muscular Atrophy II (S46.004). Neurology Apr 2018, 90 (15 Supplement) S46.004
14. Lexicomp Online. (2023, April). AHFS DI. Nusinersen. Retrieved August 2023 from Lexicomp Online with AHFS.
15. MICROMEDEX Healthcare Series. Drugdex Evaluations. (2022, November). Nusinersen. Retrieved August 2023 from MICROMEDEX Healthcare Series.
ORIGINAL EFFECTIVE DATE: 3/30/2017
MOST RECENT REVIEW DATE: 1/1/2024
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Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
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