Criteria for Prior Authorization — Spinal Muscular Atrophy (SMA) Agents
Customize your policy alerts
Sign up for all Kansas Department of Health and Environment policy alerts
Know when Kansas Department of Health and Environment releases new policies or updates existing guidance.
Monitor payer policy activity
Defines prior authorization requirements for nusinersen (Spinraza), onasemnogene abeparvovec (Zolgensma), and risdiplam (Evrysdi) for Kansas Medicaid, including initial and renewal approval criteria, dosing limits, and documentation requirements.
No material clinical or coverage changes in this revision.
Coverage Criteria for SMA Therapies
Initial Approval
Covered when ALL of the following are met
See Table 1 for age and dosing limits.
Agent-specific Initial Criteria
AND for agent-specific requirements
Provider must document SMN2 copies ≥2 per initial criteria (when applicable).
Provider must document SMN2 copies ≥2 per initial criteria (when applicable).
Zolgensma is a one-time gene therapy; see Zolgensma Authorization for authorization length.
Renewal Approval (Nusinersen or Risdiplam)
Covered when ALL of the following are met
Length of renewal approval: 12 months.
Zolgensma Authorization
Patient must meet Zolgensma-specific initial criteria (age <2 years, bi-allelic SMN1 mutation, absence of c.859G>C on exon 7, anti-AAV9 ≤1:50 ELISA, no advanced SMA).
Zolgensma (onasemnogene) is excluded for patients who meet the policy definition of advanced SMA — defined as complete paralysis of limbs or permanent ventilator dependence. Baseline laboratory testing demonstrating anti‑AAV9 antibody titers > 1:50 by ELISA also precludes eligibility. Zolgensma is a one‑time gene replacement infusion and must not be prescribed concurrently with other SMA disease‑modifying therapies; prior receipt of Zolgensma likewise disqualifies repeat administration.
Concurrent use of more than one SMA disease‑modifying agent is not permitted. Requests that propose combination therapy with two or more of Evrysdi (risdiplam), Spinraza (nusinersen), or Zolgensma (onasemnogene) will not meet criteria. If a patient is to receive Zolgensma as replacement therapy, it must not be given concurrently with Evrysdi or Spinraza.
Drugs, Genetic and Lab Criteria
| Nusinersen (Spinraza®) | Antisense Oligonucleotide — treatment of spinal muscular atrophy; dosing per Table 1 |
| Onasemnogene (Zolgensma®) | Adeno-associated virus vector-based gene therapy — treatment of spinal muscular atrophy; dosing per Table 1 |
| Risdiplam (Evrysdi®) | SMN2-directed RNA splicing modifier — treatment of spinal muscular atrophy; dosing per Table 1 |
Provider Requirements and Authorization Notes
Prior authorization required for all SMA agents; agent‑specific initial requirements and limits
Prior authorization is required for all current and future dose forms of nusinersen (Spinraza), onasemnogene (Zolgensma), and risdiplam (Evrysdi). Initial approval requires the agent be approved for the requested indication, age, and dose per Table 1 and that the prescriber be a neurologist with SMA expertise. For Evrysdi or Spinraza, the provider must submit documentation that the patient has ≥ 2 copies of the SMN2 gene. Zolgensma is authorized as a single infusion (1.1 x 10^14 vg/kg) with a 1-month authorization length; reauthorization is not permitted (one infusion per lifetime).
- PA required for all current and future dose forms of nusinersen, onasemnogene, and risdiplam. [[cited]]
- Initial approval: agent must be approved for indication, age, and dose in Table 1 and prescribed by or in consultation with a neurologist experienced in SMA. [[cited]]
- For Evrysdi or Spinraza: submit SMN2 copy number documentation (≥ 2 copies). [[cited]]
- Zolgensma: single infusion 1.1 x 10^14 vg/kg; authorization length 1 month; one infusion lifetime. [[cited]]
No concurrent combination therapy; switching to Zolgensma prohibits concurrent use
Concurrent combination therapy with more than one SMA agent (Evrysdi, Spinraza, Zolgensma) is not permitted. If Zolgensma is given to replace Evrysdi or Spinraza, it must not be prescribed concurrently as dual therapy.
- Concurrent combination therapy with more than one of Evrysdi, Spinraza, or Zolgensma is excluded and will not meet criteria.
- Zolgensma must not be prescribed concurrently with Evrysdi or Spinraza when replacing those therapies.
Provide baseline and follow‑up motor assessments and SMN2 copy documentation
Submit baseline and follow-up objective motor function assessments and documentation of motor milestones as part of the PA request and for renewals. For Evrysdi or Spinraza initial requests, include SMN2 copy number documentation (≥ 2 copies).
- Baseline documentation: HINE, HFMSE, ULM/RULM, CHOP-INTEND, MFM32, or current motor milestone status (e.g., ability to sit or ambulate).
- For renewals and monitoring: submit recent post-treatment results (<1 month) showing specified point increases or prescriber attestation of new motor milestone/maintained function.
- For Evrysdi/Spinraza initial requests: include SMN2 copy number ≥ 2.
Denial risks: genetic confirmation, permanent ventilation, and Zolgensma-specific exclusions
Requests may be denied if the diagnosis is not confirmed by SMN1 gene mutation or deletion, if the patient is on permanent ventilation (≥ 16 hours/day for > 21 days), or if Zolgensma criteria are not met (age ≥2 years, presence of advanced SMA, absence of required genetic testing or anti‑AAV9 antibody titer > 1:50).
- Diagnosis must be confirmed by SMN1 gene mutation or deletion (e.g., homozygous deletion of exon 7 or compound heterozygous mutation).
- Patient must not be on permanent ventilation (≥ 16 hours/day for > 21 days) in the absence of an acute reversible event or tracheostomy.
- Zolgensma-specific denial risks: patient must be < 2 years, must not have advanced SMA (complete limb paralysis or permanent ventilator dependence), must have genetic testing confirming absence of c.859G>C on exon 7, and must have anti‑AAV9 antibody titers ≤ 1:50 by ELISA; failure to meet these will result in denial.
Background
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder caused by bi‑allelic SMN1 mutation or deletion. Disease‑modifying therapies available include an antisense oligonucleotide (nusinersen/Spinraza), a gene replacement therapy delivered by AAV9 vector (onasemnogene/Zolgensma), and an SMN2 splicing modifier (risdiplam/Evrysdi). Genetic confirmation of SMA (SMN1 mutation or deletion) and baseline motor function assessments are required to determine eligibility and to monitor response to therapy; additional agent‑specific requirements (for example, SMN2 copy number for Evrysdi/Spinraza and anti‑AAV9 antibody titer and age limits for Zolgensma) apply.
Definitions and Key Terms
OpenPayer is powered by Trek Health's payer performance platform. Trek continuously ingests, validates, and normalizes Transparency in Coverage data alongside payer policies and other commercial payer data to create a structured payer intelligence foundation. OpenPayer uses this foundation to deliver personalized search results, dynamically generated policy pages, and tailored policy monitoring based on each user's payers, specialties, billing codes, and areas of interest. The same intelligence powers broader payer performance workflows, including reimbursement benchmarking, contract evaluation, payer negotiations, and financial decision-making.