Spinraza (nusinersen)

POLICY NUMBER

L.5.01.512

Spinraza (nusinersen)

Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

DESCRIPTION

Spinraza (nusinersen) is an antisense oligonucleotide (ASO) designed to treat spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Nusinersen increases exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.

Spinraza (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Dosage

POLICY

Spinraza (nusinersen) does not require prior authorization. However, all claims will be reviewed for medical necessity based on medical policy guidelines outlined below.

Initial Criteria

Spinraza (nusinersen) may be considered medically necessary when ALL of the following criteria are met:

1. The individual has a documented diagnosis of spinal muscular atrophy confirmed by one of the following:

   1. Homozygous deletion of SMN1 gene;

   2. Homozygous absence of SMN1 gene due to gene conversion (i.e. SMN1 gene conversion to SMN2 gene); OR

   3. Compound heterozygote mutation of SMN1 gene;

2. The prescriber has provided documentation of ONE of the following:

   1. The individual has no more than 3 copies of SMN2 gene as determined by genetic testing; OR

   2. The individual had onset of SMA symptoms prior to 6 months of age;

3. The prescriber has provided documentation of ONE of the following:

   1. For individuals with SMA Type I and II, documentation showing symptoms onset prior to 21 months of age; OR

   2. For individuals with SMA Type III, documentation showing symptoms onset after 18 months of age;

4. The prescriber has provided documentation of the baseline exam of at least one of the following exams (based on individual's age and motor ability) to establish baseline motor ability:

   1. Hammersmith Infant Neurological Exam Part 2 (HINE-2) [infant to early childhood];

   2. Hammersmith Functional Motor Scale Expanded (HFMSE);

   3. Upper Limb Module (ULM) Test [non-ambulatory]; OR

   4. Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND);

5. The individual has not previously received gene replacement therapy for the treatment of SMA;

6. The individual is not receiving concomitant chronic survival motor neuron (SMN) modifying therapy [e.g., Evrysdi (risdiplam)];

7. The prescriber is a specialist or has consulted with a specialist in an area of the individual's diagnosis (i.e., neurologist, geneticist);

8. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

9. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Renewal Criteria:

Spinraza (nusinersen) may be approved for RENEWAL when ALL of the following criteria are met:

1. The individual has been previously approved for therapy with the requested agent through BCBSMS PA process;

2. The individual has documented (within the previous 30 days) positive clinical response from pretreatment baseline status as demonstrated by at least one of the following:

   1. HINE-2 milestones: BOTH of the following:

      1. ONE of the following:

         1. Improvement or maintenance of previous improvement of at least 2 point (or maximal score) increase in ability to kick; OR

         2. Improvement or maintenance of previous improvement of at least 1 point increase in any other HINE-2 milestone (e.g., head control, rolling, sitting, crawling, etc.), excluding voluntary grasp; AND

      2. ONE of the following:

         1. Improvement or maintenance of previous improvement in more HINE motor milestones than worsening, from pretreatment baseline (net positive improvement); OR

         2. Individual has achieved and maintained any new motor milestones when otherwise unexpected to do so (e.g., sit unassisted, walk);

   2. HFMSE: ONE of the following:

      1. Improvement or maintenance of previous improvement of at least a 3 point increase in score from pretreatment baseline; OR

      2. Individual has achieved and maintained any new motor milestones when otherwise unexpected to do so;

   3. ULM: ONE of the following:

      1. Improvement or maintenance of previous improvement of at least a 2 point increase in score from pretreatment baseline; OR

      2. Individual has achieved and maintained any new motor milestones when otherwise unexpected to do so; OR

   4. CHOP INTEND: ONE of the following:

      1. Improvement or maintenance of previous improvement of at least a 4 point increase in score from pretreatment baseline; OR

      2. Individual has achieved and maintained any new motor milestones when otherwise unexpected to do so;

3. The prescriber is a specialist or has consulted with a specialist in an area of the individual's diagnosis (i.e., neurologist, geneticist);

4. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

5. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage. 

Spinraza (nusinersen) is considered not medically necessary for all other indications.

POLICY EXCEPTIONS

State Health Plan (State and School Employees) Participants

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

A.  consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

B.  appropriate with regard to standards of good medical practice; and

C.  not solely for the convenience of the Member, his or her Provider; and

D.  the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

BCBSMS may request medical records for determination of medical necessity. When medical records are requested, letters of support and/or explanation are often useful, but are not sufficient documentation unless all specific information needed to make a medical necessity determination is included.

POLICY HISTORY

07/01/2017: New policy added.

08/15/2017: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

12/22/2017: Code Reference section updated to add new 2018 HCPCS code J2326.

07/01/2018: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Sources updated.

10/01/2019: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Table in policy description updated. Policy statement criteria for initial approval updated regarding documentation of symptoms. For initial approval, added note that approval includes the 4 loading doses and 1 maintenance dose for the remainder of the 6 months. Total of 5 doses for the initial 6 months. Sources updated.

07/13/2021: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Policy description updated regarding indications for Spinraza (nusinersen) and to revise dosage table. Policy section updated to revise initial and renewal criteria. Updated length of approval for renewal. Policy Exceptions updated. Policy Guidelines updated to change "Nervous/Mental Conditions" to "Mental Health Disorders" and "Medically Necessary" to "medical necessity." Added information regarding BCBSMS request for medical records. Sources updated. Code Reference section updated to change "Covered Codes" to "Medically Necessary Codes."

09/09/2022: Policy section updated to remove length of approvals for initial and renewal criteria.

02/01/2023: Medically necessary criteria revised to state the following: The prescriber has provided documentation of ONE of the following: A) The member has no more than 3 copies of SMN2 gene as determined by genetic testing; B) The member had onset of SMA symptoms prior to 6 months of age. Policy statement updated to change "provider" to "prescriber." Sources updated.

07/01/2023: Policy Exceptions updated regarding State Health Plan (State and School Employees) Participants.

06/03/2025: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy statement criteria updated to change "member" to "individual." Sources updated.

SOURCE(S)

1. Bodamer, O. Spinal muscular atrophy. UpToDate. Last accessed April 8, 2025. https://www.uptodate.com/contents/spinal-muscular-atrophy

2. Chiriboga CA, Swoboda KJ, Darras BT, et al. Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy. Neurology. 2016 Mar 8;86(10):890-7.

3. Glanzman AM, O'Hagen JM, McDermott MP, et al. Validation of the Expanded Hammersmith Functional Motor Scale in spinal muscular atrophy type II and III. J Child Neurol. 2011;26(12):1499-507.

4. Haataja L, Mercuri E, Regev R, et al. Optimality score for the neurologic examination of the infant at 12 and 18 months of age. J Pediatr. 1999 Aug;135(2 Pt 1):153-61.

5. Spinraza prescribing information. Biogen. April 2024. Last accessed April 2025.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

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