CurrentPremera BluecrossPolicy 5.01.28

Pharmacotherapy of Spinal Muscular Atrophy (SMA)

Defines medical necessity, benefit routing, dosing limits, and documentation requirements for Evrysdi (risdiplam), Spinraza (nusinersen), and Zolgensma (onasemnogene abeparvovec-xioi) for individuals with SMA and specifies when use is investigational.

Policy Summary

PayerPremera Bluecross

PolicyPharmacotherapy of Spinal Muscular Atrophy (SMA)

Policy CodePolicy 5.01.28

Change TypeNo material clinical or coverage changes

Effective Date

Next Review Date

Key ActionObtain prior authorization and document genetic testing showing bi-allelic SMN1 mutations and SMN2 copy number per the medical necessity criteria.

SourceLink

3FDA‑approved SMA therapies addressed

≤2 yrsAge limit for Zolgensma infusion

Up to 12 moApproval period (Evrysdi/Spinraza)

One-timeZolgensma administration

C9309HCPCS/J-codes listed

Liver BBW

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Coverage Criteria

Evrysdi (risdiplam) — Initial Therapy

Evrysdi (risdiplam) may be considered medically necessary when ALL of the following are met

Evrysdi initial criteria

Dosing: 0.15 mg/kg per day for individuals <2 months of age; 0.2 mg/kg per day for individuals 2 months to <2 years of age; 0.25 mg/kg per day for individuals ≥2 years of age and weighing <20 kg; 5 mg per day for individuals ≥2 years of age and weighing ≥20 kgexact doses

Prescriber attestation/documentation: Initial approval requires chart notes documenting the diagnosis and genetic testing documenting bi-allelic SMN1 mutations and copies of SMN2 gene

Spinraza (nusinersen) — Initial Therapy

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

Spinraza initial criteria: Diagnosis of SMA Type 1, 2, or 3 attributed to bi-allelic SMN1 mutations documented by genetic testing; has not received Zolgensma (onasemnogene abeparvovec-xioi); medication is prescribed by a neurologist with expertise treating SMA; Spinraza will not be used in combination with Evrysdi (risdiplam); recommended dosing schedule: 12 mg per administration with 4 loading doses (first 3 at 14-day intervals, 4th loading dose 30 days after the 3rd) followed by maintenance dosing every 4 monthsdosing schedule

Prescriber attestation/documentation: Initial approval requires chart notes documenting the diagnosis and genetic testing documenting bi-allelic SMN1 mutations and copies of SMN2 gene

Zolgensma (onasemnogene abeparvovec-xioi) — Initial Therapy

Zolgensma (onasemnogene abeparvovec-xioi) may be considered medically necessary when ALL of the following are met

Zolgensma initial criteria: Individual is aged less than 2 years at time of infusion; diagnosis of SMA attributed to bi-allelic SMN1 mutations documented by genetic testing; documented SMN2 copy number of 4 or fewer; individual does not have advanced SMA; baseline anti-AAV9 antibody titer is ≤1:50; medication is prescribed by a neurologist with expertise treating SMA; Zolgensma is administered as a one-time intravenous infusion at a dose of 1.1 x 10^14 vector genomes per kg over 60 minutes; prescriber attests to providing clinical outcome information within the appropriate provider portal as requestedage<2, SMN2≤4, anti-AAV9≤1:50

Pre-infusion requirements: Document baseline anti-AAV9 antibody level and baseline liver function; plan for peri-infusion corticosteroid administration per product guidance and monitoring

Continuation Therapy / Re-authorization

Re-authorization criteria for Evrysdi and Spinraza

Re-authorization: Medical records must document positive clinical response to therapy as evidenced by improvement in functional measures appropriate to the individual's current abilities (for example: ambulation, arm strength, or pulmonary function)

Documentation: Re-authorization requests must include chart notes documenting progress and the functional measures used to evaluate response

The policy lists specific exclusions for Evrysdi (risdiplam) and Spinraza (nusinersen). Use of these agents is considered investigational when used for SMA not attributed to bi‑allelic SMN1 mutations, for SMA type 4 (adult‑onset), when used after Zolgensma infusion, or when Evrysdi and Spinraza are used in combination.

Zolgensma (onasemnogene abeparvovec‑xioi) is considered investigational for individuals with a documented SMN2 copy number of 5 or more; uses not outlined in the policy are categorized as investigational.

In the Phase I onasemnogene abeparvovec (Zolgensma) trial, subjects were excluded if they had preexisting anti‑AAV9 antibody titers greater than 1:50, supporting use of an anti‑AAV9 titer threshold in eligibility assessments.

This medical policy does not apply to Medicare Advantage plans. Coverage determinations remain subject to the member's benefit plan limits and conditions.

All other uses of Evrysdi, Spinraza, and Zolgensma that are not explicitly described as covered in this policy are considered investigational / not medically necessary.

Initial Therapy — Eligibility and Dosing

Initial therapy — Evrysdi, Spinraza, Zolgensma

Initial therapy eligibility and dosing for Evrysdi, Spinraza, and Zolgensma

Evrysdi initial: See Evrysdi criteria: diagnosis of SMA Types 1–3 with bi-allelic SMN1 mutations, no prior Zolgensma, prescriber expertise, no combination with Spinraza, weight/age dosing limits as specified

Spinraza initial: See Spinraza criteria: diagnosis of SMA Types 1–3 with bi-allelic SMN1 mutations, no prior Zolgensma, prescriber expertise, no combination with Evrysdi, loading and maintenance dosing schedule as specified

Zolgensma initial: See Zolgensma criteria: age <2 years at infusion, SMN2 copy number ≤4, baseline anti-AAV9 antibody ≤1:50, not advanced SMA, one-time IV infusion at 1.1 x 10^14 vg/kg over 60 minutes, prescriber attestation to submit outcomesage<2, SMN2≤4, anti-AAV9≤1:50

Continuation / Re-authorization Criteria

Re-authorization — Evrysdi and Spinraza

Re-authorization requirements for Evrysdi and Spinraza

Evrysdi/Spinraza re-authorization: Must document positive clinical response with improvement in functional measures appropriate to the individual's current abilities (e.g., ambulation, arm strength, pulmonary function)

Approval duration: Non-formulary exception or re-authorization approvals for Evrysdi and Spinraza may be granted for up to 12 months when criteria are met

Step Therapy and Sequencing

Restriction	Policy statement / details	Coverage label
Concurrent use of Evrysdi (risdiplam) and Spinraza (nusinersen)	Evrysdi will not be used in combination with Spinraza; Spinraza will not be used in combination with Evrysdi.	Not allowed
Prior receipt of Zolgensma (onasemnogene abeparvovec-xioi)	Individuals who have received Zolgensma are ineligible to initiate Evrysdi or Spinraza per policy exclusions.	Not allowed
Zolgensma as single-dose gene therapy and subsequent therapy	Zolgensma is administered as a one-time infusion; use of Evrysdi or Spinraza after Zolgensma infusion is considered investigational and would trigger denial.	Not allowed (post‑Zolgensma use)

Assessment	Key findings cited by policy	Influence on step/coverage decisions
ICER (Institute for Clinical and Economic Review) comparative assessments	ICER published a final report (April–May 2019) appraising nusinersen and onasemnogene abeparvovec-xioi; concluded high certainty of substantial net health benefit for infantile-onset SMA for both agents, but noted insufficient evidence for many other SMA subtypes and for direct comparisons.	May guide payers to differentiate coverage by age/onset and to recognize evidence gaps when considering sequencing; supports cautious, criteria‑based access rather than automatic interchangeability.
ICER conclusions for nusinersen	For infantile-onset SMA ICER rated nusinersen as providing a substantial net health benefit; later-onset and presymptomatic conclusions indicated moderate certainty of at least a small net benefit, with limitations in generalizability and long-term data.	Supports coverage for nusinersen in infantile and later-onset SMA with documentation; indicates need for prescriber expertise and monitoring of real‑world outcomes when applied beyond trial populations.
ICER appraisal of onasemnogene abeparvovec-xioi (Zolgensma)	ICER based its assessment primarily on the Phase I dose-finding study; concluded high certainty of substantial net health benefit for infantile-onset SMA but insufficient evidence for many other SMA types and for comparisons to nusinersen; highlighted uncertainty about long-term durability and some patients later received nusinersen.	Informs policy rationale for restricting Zolgensma to age <2 years with SMN2 ≤4 and for excluding/limiting sequential or combined therapy until more comparative data are available.

Documented prior therapies / sequencing	Policy excerpt / history	Implication for current policy
Explicit sequencing requirements in current excerpt	The excerpt does not specify a stepwise sequence of prior therapies beyond requiring that individuals have not received Zolgensma to be eligible for Evrysdi or Spinraza; documented prior therapies or detailed sequencing requirements are not stated in this excerpt.	Not specified
Policy history showing additions of therapies and coding changes	History documents addition of Spinraza, Zolgensma, and Evrysdi criteria and multiple coding updates (coding additions/terminations dated 2017–2026), but does not add explicit sequencing rules in the policy text.	Contextual — may indicate iterative policy updates rather than defined step tiers
Operational requirement implied by history and exclusions	While sequencing is not prespecified, the policy explicitly excludes use of Evrysdi or Spinraza after Zolgensma and prohibits concurrent Evrysdi+Spinraza, implying these sequencing constraints must be documented at authorization.	Enforceable constraints present (post‑Zolgensma and concurrent use)

Coding

HCPCS/J-codes listedHCPCS

C9309	Injection, onasemnogene abeparvovec-brve, per treatment (new code effective 04/01/26)
J2326	Injection, nusinersen (Spinraza), 0.1 mg
J3399	Injection, onasemnogene abeparvovec-xioi, per treatment, up to 5x10^15 vector genomes (Zolgensma)

Policy coding updatesHCPCS

C9309

new HCPCS code added effective April 1, 2026

Historical HCPCS codes mentionedHCPCS

C9489	previous HCPCS code (terminated 1/1/18)
J2326	HCPCS code added 1/1/18
J3399	HCPCS code added 02/01/21
J3590	HCPCS code added 07/01/19 then removed 02/01/21
J3490	HCPCS code removed 05/01/18

inv-13: Anti-AAV9 antibody titer threshold

Anti-AAV9 antibody titer thresholdBaseline anti-AAV9 (AAV9) antibody levels are at most 1:50 for Zolgensma eligibility; Phase I trial excluded subjects with titers >1:50.

Clinical implicationDocument baseline anti-AAV9 antibody titer prior to infusion; values >1:50 are a contraindication per eligibility criteria and prior trial exclusions.

Source policy actionPre-infusion antibody testing is required and results must be included in authorization documentation.

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Zolgensma, Evrysdi, and Spinraza per policy. For Zolgensma: prescriber must attest to providing clinical outcome information within the appropriate provider portal as requested by the Company plan.

Prior authorization required for Zolgensma, Evrysdi, and Spinraza.
Prescriber must attest to submitting clinical outcome data for Zolgensma via the provider portal.

Documentation Required

Pre‑infusion Labs and Antibody Testing

Before Zolgensma infusion, obtain baseline liver function testing (AST, ALT, total bilirubin, prothrombin time) and anti-AAV9 antibody titers. Anti-AAV9 antibody titer must be ≤ 1:50. Provide documentation of pre-infusion labs and antibody testing with the authorization request.

Obtain AST, ALT, total bilirubin, and prothrombin time prior to infusion.

Site of Care and Administration

Note

Zolgensma limited to certified treatment centers

Zolgensma is available only at certified treatment centers and must be administered as a single IV infusion in a monitored setting with a corticosteroid plan and liver monitoring.

Arrange treatment at a certified treatment center prior to authorization and infusion scheduling.
Ensure the infusion is performed in a monitored setting with the planned corticosteroid regimen documented.

Note

Zolgensma administered as single IV infusion with corticosteroid management

Onasemnogene abeparvovec is administered as a single intravenous infusion over approximately 60 minutes and requires pre‑ and post‑infusion corticosteroid management and monitoring.

Dosing is 1.1 x 10^14 vector genomes per kg over ~60 minutes per product labeling.
Document corticosteroid administration plan and liver monitoring in the medical record.

Definitions

inv-31: Genetic diagnosis requirement for SMA therapies (bi-allelic SMN1 mutations)

Required genetic diagnosisDiagnosis of SMA must be attributed to bi-allelic mutations of the SMN1 gene documented by genetic testing.

Documentation neededChart notes must include genetic testing demonstrating bi-allelic SMN1 mutations for initial approval and authorization.

Coverage scopeThis genetic requirement applies to Evrysdi, Spinraza, and Zolgensma initial therapy criteria.

inv-32: SMN2 copy number used to stratify severity and eligibility for Zolgensma (<=4 copies required)

SMN2 copy-number eligibilityZolgensma eligibility requires documented SMN2 copy number of 4 or fewer (≤4 SMN2 copies).

Severity stratificationSMN2 copy number is used to stratify SMA severity and informs treatment decisions (higher SMN2 copies generally associate with milder phenotypes).

Background

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder caused by bi‑allelic SMN1 mutations leading to insufficient survival motor neuron (SMN) protein, progressive motor neuron loss, and muscle weakness. Disease severity correlates inversely with SMN2 copy number and age of onset. Gene‑replacement therapy with onasemnogene abeparvovec (Zolgensma) delivers an SMN1 transgene via an AAV9 vector as a one‑time IV infusion and carries specific safety considerations including liver injury risk observed in early trials; the Phase I study excluded subjects with anti‑AAV9 antibody titers > 1:50 and employed peri‑infusion corticosteroids and liver monitoring in response to aminotransferase elevations. Coverage and benefit determinations under this policy are subject to the member benefit plan and do not apply to Medicare Advantage.

Evidence and Trials

inv-36: SMA therapies evidence — trial citations and study types

Zolgensma pivotal trialsPhase I (START) and subsequent Phase III trials (STR1VE, STR1VE-EU, SPR1NT) reported open‑label single-arm efficacy with milestone attainment and ventilation avoidance in treated infants.

Nusinersen pivotal trialsENDEAR (infantile-onset RCT) showed reduction in death or permanent ventilation and improved motor milestones; later-onset RCTs and extension studies reported sustained benefits.

Risdiplam pivotal trialsSUNFISH (phase II/III randomized placebo-controlled for types 2/3) and FIREFISH (type 1 open-label) reported motor function improvements and CHOP-INTEND increases in infants; JEWELFISH and RAINBOWFISH contribute additional cohort and presymptomatic data.

Pivotal evidence summaries (alternate placement for trial outcome highlights)

Efficacy summaries from pivotal and major trials (alternate placement highlighting trial outcomes)

SUNFISH (risdiplam):

Revision History

2025policy clarificationLatest

Clarified that non‑formulary exception review authorizations for all drugs listed may be approved up to 12 months and that medications are subject to the product's FDA dosing and administration; added requirement that Zolgensma prescribers attest to providing clinical outcome information in the provider portal.

2018-03-30literature review

Conducted literature search through 3/30/2018 and added reauthorization period and criteria after finding no new clinical data requiring changes to coverage criteria.

Policy Summary

PayerPremera Bluecross

PolicyPharmacotherapy of Spinal Muscular Atrophy (SMA)

Policy CodePolicy 5.01.28

Change TypeNo material clinical or coverage changes

Effective Date

Next Review Date

Key ActionObtain prior authorization and document genetic testing showing bi-allelic SMN1 mutations and SMN2 copy number per the medical necessity criteria.

SourceLink

On This Page

Pivotal evidence summaries

Efficacy summaries from pivotal and major trials

Risdiplam (SUNFISH/FIREFISH/JEWELFISH/RAINBOWFISH): SUNFISH (Types 2/3) showed mean MFM-32 improvement and statistically significant differences vs placebo at 12 months (MFM-32 and Revised Upper Limb Module); FIREFISH (Type 1) open-label part showed CHOP-INTEND increases with a median increase of 16 points in part one (N=21); JEWELFISH demonstrated increased SMN protein in a small subgroup; RAINBOWFISH presymptomatic infants reached major motor milestones at 12 months

Nusinersen (ENDEAR and other studies): ENDEAR showed a 29% reduction in risk of death or permanent ventilation and improved motor milestones versus sham control; presymptomatic and other studies demonstrated milestone attainment and maintenance in Types 2 and 3

Onasemnogene abeparvovec (Phase I/START): Phase I open-label study in infants with SMA1 (two SMN2 copies) showed increases in CHOP-INTEND, milestone attainment (sitting, rolling, some walking in cohort 2), and avoidance of permanent ventilation through follow-up; subjects with anti-AAV9 titers >1:50 were excluded

Document anti‑AAV9 antibody titer ≤ 1:50 before infusion.

Denial Risk

Zolgensma: Pre/Post Infusion Liver Monitoring

Per FDA boxed warning and policy safety evidence, administer corticosteroids before and after Zolgensma infusion and monitor liver function for at least 3 months post‑infusion. Document post‑infusion liver monitoring and steroid administration; omission of documented monitoring may lead to denial.

Administer corticosteroids per prescribing information before and after infusion.
Monitor liver function (AST, ALT, bilirubin, PT) for at least 3 months after infusion.
Lack of documented post‑infusion liver monitoring may result in denial.

Prior Authorization

Authorization Duration and Dosing Adherence

Zolgensma is a one‑time infusion; approvals for Zolgensma reflect one‑time dosing. Non‑formulary exception reviews and other authorizations for Evrysdi and Spinraza may be approved up to 12 months. Ensure dosing adheres to FDA product labeling and the policy's dose limits; deviations require non‑formulary justification.

Zolgensma = one‑time infusion (single authorization).
Evrysdi and Spinraza non‑formulary or standard authorizations may be approved for up to 12 months.
Dosing must follow FDA labeling and policy dose limits; non‑formulary exceptions require documentation.

Billing Rule

Prior Authorization and Coding

All requests should include appropriate coding (medical benefit HCPCS/CPT) and supporting documentation. Be aware of recent coding updates (eg, HCPCS C9309 added 4/1/2026) and include the correct code for the billed service.

Include appropriate HCPCS/CPT codes on the claim and authorization (verify current code list).
Note coding updates such as HCPCS C9309 effective 04/01/2026.

Prior Authorization

Non‑Formulary Exception and Dosing Adherence

Non‑formulary exception approvals require attestation of dosing adherence and may be granted up to 12 months. Provide rationale and documentation for any dosing or administration that deviates from FDA prescribing information.

Non‑formulary exception reviews may be approved up to 12 months.
Include rationale and documentation when dosing deviates from FDA prescribing information.

Denial Risk

Benefit Limitations May Trigger Denial

Benefit limitations in the member's contract may affect coverage; check the member's benefit booklet or contact member services. Lack of applicable benefits or plan exclusions may lead to denial of the request.

Verify member-specific benefit limitations before authorization.
Denial may occur if member benefits do not cover the requested therapy.

Documentation Required

Re‑authorization Documentation

Re‑authorization for Evrysdi and Spinraza requires chart notes documenting clinical progress and positive response to therapy (eg, improvement in ambulation, arm strength, or pulmonary function). Future re‑authorization of Zolgensma beyond the one‑time infusion is considered investigational.

Submit chart notes documenting functional improvements for re‑authorization of Evrysdi or Spinraza.
Zolgensma re‑authorization beyond one infusion is investigational and not routinely approved.

Documentation Required

Zolgensma Outcome Attestation

Prescriber must attest (via provider portal) to providing clinical outcome information for Zolgensma at the requested cadence; include outcome attestation with the authorization.

Attestation to submit clinical outcomes for Zolgensma is required.
Include outcome attestation in the authorization request.

Step Therapy

Sequential/Combination Therapy Restrictions

Policy restricts combination or sequential use: Evrysdi and Spinraza should not be used together, and Evrysdi or Spinraza use after Zolgensma is considered investigational. Decisions about sequencing may be informed by comparative evidence but evidence on combinations/sequence is limited.

Evrysdi and Spinraza must not be used in combination.
Evrysdi or Spinraza after Zolgensma is considered investigational.
Comparative evidence is limited and may inform but not definitively direct step decisions.

Note

Comparative Evidence May Inform Step Decisions

Comparative effectiveness reports (eg, ICER) note limited evidence for some comparisons and highlight uncertainty regarding combination/sequence therapy; use available comparative evidence to inform step decisions when applicable.

Refer to ICER and published comparative evidence when making step or sequencing decisions.
Recognize uncertainty due to limited comparative data, especially for onasemnogene vs nusinersen.

Step Therapy

Step Therapy Not Specified in This Excerpt

The policy text does not specify a formal step‑therapy sequence between Evrysdi and Spinraza in this excerpt; follow the documented medical necessity criteria and individual clinical circumstances when selecting therapy.

No explicit step‑therapy order between Evrysdi and Spinraza is specified in this policy excerpt.
Select therapy based on medical necessity criteria and clinician judgment.

Note

Spinraza administered intrathecally per FDA dosing schedule

Nusinersen (Spinraza) is administered intrathecally per product dosing schedule (4 loading doses followed by maintenance every 4 months); billing and administration must reflect intrathecal delivery.

Loading: first 3 doses at 14‑day intervals; 4th loading dose 30 days after the 3rd; maintenance every 4 months.
Document intrathecal administration details in the medical record for billing and authorization.

Billing Rule

Follow FDA dosing/administration at infusion centers and hospital outpatient settings

Infusion center and hospital outpatient administrations must follow the product's FDA dosing and administration prescribing information when delivering these therapies.

Ensure infusion protocols and monitoring align with FDA labeling for each product.
Document adherence to FDA dosing and administration when submitting claims or authorizations.

Investigational use noteZolgensma use for individuals with 5 or more SMN2 copies is considered investigational per policy exclusions.

inv-33: Risdiplam / Evrysdi definition and trial context

DefinitionRisdiplam (Evrysdi) is an oral SMN2 splicing modifier that increases exon 7 inclusion in SMN2 transcripts to raise functional SMN protein levels.

Trial contextRisdiplam was evaluated in SUNFISH (types 2/3), FIREFISH (type 1), JEWELFISH (previously treated patients), and RAINBOWFISH (presymptomatic infants) with reported motor function improvements in some cohorts.

Regulatory statusEvrysdi is FDA‑approved for treatment of SMA (policy discusses dosing and coverage under pharmacy benefit).

inv-34: Nusinersen / Spinraza definition

DefinitionNusinersen (Spinraza) is an antisense oligonucleotide directed at SMN2 that increases exon 7 inclusion to produce more functional SMN protein.

AdministrationAdministered intrathecally with a recommended dosage of 12 mg per administration: 4 loading doses (first 3 at 14-day intervals, 4th at 30 days after 3rd) then maintenance every 4 months.

Coverage constraintsSpinraza must not be used in combination with Evrysdi and is managed under the medical benefit per policy criteria.

inv-35: Onasemnogene abeparvovec / Zolgensma definition

DefinitionOnasemnogene abeparvovec (Zolgensma) is an AAV9-based IV gene replacement therapy that delivers a functional SMN1 copy to patient cells as a one-time infusion.

Dosing & administrationRecommended dose is 1.1 x 10^14 vector genomes per kg administered IV over ~60 minutes; requires administration at certified treatment centers.

Safety considerationsZolgensma carries risks of hepatotoxicity; baseline liver assessment, corticosteroid administration, and post-infusion liver monitoring are required.

inv-36: SMA therapies evidence — referenced literature

Key randomized/phase trials citedNusinersen: ENDEAR (infantile-onset RCT) and later-onset RCTs; Onasemnogene abeparvovec: Phase I (START) and Phase III (STR1VE, STR1VE-EU, SPR1NT); Risdiplam: SUNFISH (phase II/III), FIREFISH, JEWELFISH, RAINBOWFISH.

Evidence typesCited literature includes randomized controlled trials, open-label phase 1 and 3 studies, registries, and regulatory clinical reviews informing efficacy and safety conclusions.

Guidance and assessmentsPolicy references ICER and NICE assessments as comparative-effectiveness resources alongside trial data.

inv-36: SMA therapies evidence (additional chunk reference)

Additional referencesSelected additional citations include RESTORE registry design, risdiplam comparative analyses versus historical controls, and multiple risdiplam phase 2/3 publications (NEJM, Lancet Neurol).

Ongoing researchPolicy notes ongoing and planned clinical trials (intrathecal and IV gene therapy studies, multiple risdiplam studies) and registry efforts informing long-term outcomes.

Reference rangeReference list in policy includes >70 citations covering pivotal trials, extensions, registries, and reviews.

In SUNFISH part two, risdiplam showed statistically significant improvements at 12 months versus placebo in MFM-32 and Revised Upper Limb Module scores (differences 1.55 and 1.59 respectively)

FIREFISH (risdiplam): FIREFISH part one (N=21) showed a median CHOP-INTEND increase of 16 points and ≥4-point increase in 93% of individuals at 245 days

ENDEAR (nusinersen): ENDEAR demonstrated a 29% reduction in risk of death or permanent ventilation and improved motor milestones compared to sham control

Zolgensma Phase I: Phase I single-arm study (N=15) in infants with two SMN2 copies reported milestone attainment and avoidance of permanent ventilation; subjects with anti-AAV9 antibody titers >1:50 were excluded

2017-04-01policy adoption

New policy approved (new policy effective/dated 04/01/17) adding coverage for nusinersen (Spinraza) under prescription drug section.

2022-08-01annual review

Annual review approved 07/25/2022 with update to Evrysdi (risdiplam) criteria removing the requirement that the individual be at least 2 months of age.