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)    Spinraza (Nusinersen) is approved by the FDA for the treatment of Spinal Muscular Atrophy (SMA)

2)    Initial requests for Spinraza (Nusinersen) require preauthorization to ensure the diagnosis of SMA has been confirmed by a neurologist or pediatric neurologist with the appropriate laboratory tests. 

3)    All preauthorization requests for Spinraza (Nusinersen) have a limit of only 6 months. 

4)    All requests for continuation of Spinraza (Nusinersen) beyond 6 months require a new preauthorization to ensure pediatric neurologist has documented clinically significant benefit from the therapy.

Initial Therapy***

Initial therapy with Spinraza/Nusinersen requires preauthorization and is considered medically necessary for the treatment of spinal muscular atrophy in individuals who meet criteria A AND B AND C:

1. SMA diagnosis of G12.0 (infantile spinal muscular atrophy, type 1) has been confirmed by a pediatric neurologist AND
2. Laboratory test documentation of diagnosis by either:
   1. SMA diagnostic test results confirming 0 copies of SMN1; or
   2. Molecular genetic testing of 5q SMA for any of the following:
      1. homozygous gene deletion; or
      2. homozygous conversion mutation; or
      3. compound heterozygote; AND
3. Documentation of either:
   1. Genetic testing confirming no more than 2 copies of SMN2; or
   2. SMA-associated symptoms before 6 months of age.

Continuation Therapy***

Continuation of treatment with Spinraza/Nusinersen beyond 6 months after initiation of therapy, and every 6 months thereafter, requires preauthorization and is considered medically necessary for the treatment of spinal muscular atrophy when individuals meet both criteria A AND B:

1. When initial therapy was determined to meet the above criteria, AND
2. When there is documentation (using objective findings such as the Hammersmith Functional Motor Scale or Hammersmith Infant Neurologic Exam) by a pediatric neurologist of clinically significant improvement in spinal muscular atrophy-associated symptoms (for example, progression, stabilization, or decreased decline in motor function) compared to the predicted natural history trajectory of disease.

*** If an individual meets medically necessary criteria, dosing of Spinraza/Neusinersen treatment is covered according to the Food and Drug Administration (FDA) product information label. The FDA recommends that a maintenance dose should be administered once every 4 months. As noted above, to continue therapy, medically necessary criteria requires pediatric neurologist to objectively evaluate and demonstrate Spinraza/Nusinersen`s clinical effectiveness for the treated individual every 6 months.

Codes Used In This BI:

1. Chiriboga CA, Swoboda KJ, Darras BT, et al. Results from a phase 1 study of Neusinersen (ISIS-SMN (Rx)) in children with spinal muscular atrophy. Neurology. 2016; 86(10):890-897.
2. Finkel RS, Chiriboga CA, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with Neusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016; 388(10063):3017-3026.
3. Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014; 83(9):810-817.
4. Mailman M, Heinz J, Papp A, et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet Med. 2002; 4(1):20-26.