Nusinersen (Spinraza ®)

Restricted Product(s):

• nusinersen (Spinraza®) intrathecal injection for administration by a healthcare professional

FDA Approved Use:

• For treatment of pediatric and adult patients with spinal muscular atrophy

Criteria for Medical Necessity:

The restricted product(s) may be considered medically necessary when the following criteria are met:

Initial Criteria for Approval:

1. The patient has a diagnosis of spinal muscular atrophy (SMA) [medical record documentation required]; AND 
2. The diagnosis has been confirmed by genetic testing consisting of one of the following [medical record documentation required]: a. Homozygous deletion of survival motor neuron 1 (SMN1) exon 7; OR b. Compound heterozygosity for SMN1 exon 7 deletion and small mutation; AND 
3. ONE of the following: 
   1. If the patient has SMA-associated symptoms, BOTH of the following: 
      1. The patient has two or more copies of the survival motor neuron 2 (SMN2) gene by genetic testing [medical record documentation required]; AND 
      2. The patient has SMA-associated symptom onset before 20 months of age [medical record documentation required]; OR 
   2. If the patient does not have SMA-associated symptoms (asymptomatic), the following: 
      1. The patient has two or three copies of the SMN2 gene by genetic testing [medical record documentation required]; AND 
4. The patient has been evaluated by a board-certified neurologist or pediatric neurologist experienced in the diagnosis and management of SMA prior to starting treatment with the requested agent [medical record documentation required]; AND 
5. The patient has had laboratory testing and monitoring performed at baseline, consisting of platelet count, coagulation laboratory testing, and quantitative spot urine protein testing [medical record documentation required]; AND 
6. The patient’s baseline clinical status has been assessed, documented, and submitted through at least ONE baseline assessment (based on patient age and motor ability) to establish baseline motor ability [medical record documentation required]; AND 
7. The patient has not received treatment with onasemnogene abeparvovec-xioi (Zolgensma®) or other gene therapy for the treatment of SMA [medical record documentation required]; AND
8. The patient will not be treated concurrently with risdiplam (Evrysdi™) and/or onasemnogene abeparvovec-xioi (Zolgensma ®), and any existing authorizations will be closed upon approval of nusinersen (Spinraza®)*; AND 
9. The requested agent is not being used to treat non-5q-spinal muscular atrophy disorders [medical record documentation required]; AND 
10. The prescriber is a specialist in the area of the patient’s diagnosis (i.e., board-certified neurologist or pediatric neurologist) [medical record documentation required]; AND 
11. The requested dose and dosing frequency are within FDA labeled dosing for the requested indication [medical record documentation required]. 

*Please note, receipt of any requests for alternative drugs to treat SMA (e.g., risdiplam [Evrysdi™], onasemnogene abeparvovec-xioi [Zolgensma®]) will result in closure of the nusinersen (Spinraza®) authorization.

Duration of Approval: 180 days (6 months) 

Continuation Criteria for Approval:

1. The patient was approved through Blue Cross NC initial criteria for approval; OR 
2. The patient would have met initial criteria for approval at the time they started therapy; AND 
3. The patient has demonstrated clinically significant improvement in SMA-associated symptoms (e.g., progression, stabilization, or decreased decline in motor function) during the previous treatment period with the requested agent [medical record documentation required]; AND 
4. The prescriber is a specialist in the area of the patient’s diagnosis (i.e., board-certified neurologist or pediatric neurologist) [medical record documentation required]; AND 
5. The patient has had laboratory testing and monitoring performed prior to each dose, consisting of platelet count, coagulation laboratory testing, and quantitative spot urine protein testing [medical record documentation required]; AND 
6. The patient has not received treatment with onasemnogene abeparvovec-xioi (Zolgensma®) or other gene therapy for the treatment of SMA [medical record documentation required]; AND 
7. The patient will not be treated concurrently with risdiplam (Evrysdi™) and/or onasemnogene abeparvovec-xioi (Zolgensma ®), and any existing authorizations will be closed upon approval of nusinersen (Spinraza®)*; AND 
8. The requested dose and dosing frequency are within FDA labeled dosing for the requested indication [medical record documentation required].

*Please note, receipt of any requests for alternative drugs to treat SMA (e.g., risdiplam [Evrysdi™], onasemnogene abeparvovec-xioi [Zolgensma®]) will result in closure of the nusinersen (Spinraza®) authorization.

Duration of Approval: 180 days (6 months)

References:

all information referenced is from FDA package insert unless otherwise noted below.

1. Butchbach ME. Copy number variations in the survival motor neuron genes: implications for spinal muscular atrophy and other neurodegenerative diseases. Front Mol Biosci. 2016;3:7. 
2. Chiriboga CA, Swoboda KJ, Darras BT, et al. Results from a phase 1 study of nusinersen (ISISSMN(Rx)) in children with spinal muscular atrophy. Neurology. Mar 08 2016;86(10):890 -897. 
3. Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile -onset spinal muscular atrophy. N Engl J Med. 2017;377:1723-32. 
4. Kolb SJ, Kissel JT. Spinal muscular atrophy. Neurol Clin. 2015;33(4):831. 
5. Mercuri E, Darras BT, Chiriboga CA, et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378:625-35.

Policy Implementation/Update Information

May 2022: Criteria change: Requirement of baseline clinical status and medical record documentation added.

June 2021: Criteria change: Added requirement for maximum units, dose, and dosing frequency; medical policy formatting change. Policy notification given 4/16/2021 for effective date 6/16/2021.

*Further historical criteria changes and updates available upon request from Medical Policy and/or Corporate Pharmacy. 

Non-Discrimination and Accessibility Notice

Discrimination is Against the Law

• Blue Cross and Blue Shield of North Carolina (“Blue Cross NC”) complies with applicable Federal civil rights laws and does not discriminate on the basis of race, color, national origin, age, disability, or sex. 
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• If you need these services, contact Customer Service 1-888-206-4697, TTY and TDD, call 1-800-442-7028. 
• If you believe that Blue Cross NC has failed to provide these services or discriminated in another way on the basis of race, color, national origin, age, disability, or sex, you can file a grievance with: 
  • Blue Cross NC, PO Box 2291, Durham, NC 27702, Attention: Civil Rights Coordinator- Privacy, Ethics & Corporate Policy Office, Telephone 919-765-1663, Fax 919-287-5613, TTY 1-888-291-1783
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LUS CEEV: Yog tias koj hais lus Hmoob, cov kev pab txog lus, muaj kev pab dawb rau koj. Hu rau 1-888-206-4697 (TTY: 1-800-442-7028).

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