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Drug Zolgensma® (onasemnogene abeparvovec-xioi) [AveXis Inc.]

January 2020

Therapeutic area - Spinal Muscular Atrophy (SMA)

Approval criteria

Prescriber Requirements

  • Prescriber must be a pediatric neurologist with expertise in the treatment of SMA
  • Physician attests that the patient, while under the care of the physician, will be assessed by one of the following exam scales during subsequent office visits for a period not to exceed 3 years
    • Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) scale during subsequent office visits while the patient is 2 to 3 years of age or younger; or
    • Hammersmith Functional Motor Scale Expanded (HFMSE) during subsequent office visits while the patient is 2 to 3 years of age or older
  • Prescriber agrees that documentation of follow-up patient assessment(s) including, but not necessarily limited to, serial CHOP INTEND or HFMSE assessments while the patient is under the care of the physician, will be made available to Medical Assistance upon request.
  • Physician attests that the patient has never received Zolgensma treatment.

Patient Requirements

  • Patient must be < 2 years of age; AND
  • Patient has a diagnosis of spinal muscular atrophy (SMA) confirmed by either bi-allelic deletion or dysfunctional point mutation of the SMN1 gene; AND
  • Patient must have SMA phenotype 1 confirmed by ≥ 1 of the following:
    • Patient must have 1 or 2 copies of the SMN2 gene; OR
    • Patient has 3 copies of the SMN2 gene in the absence of the c.859G>C single base substitution modification in exon 7; AND
  • Patient must have a baseline anti-AAV9 antibody titer of ≤ 1:50 measured by ELISA; AND
  • Patient does not have pre-existing hepatic insufficiency; AND
  • Patient must not have advanced disease (e.g., complete limb paralysis, permanent ventilation support); AND
  • The treatment of pre-symptomatic patients diagnosed by newborn screening who are unlikely to develop Type I or II SMA will not be approved; OR
  • The treatment of symptomatic later-onset SMA older than 2 years of age will not be approved; AND
  • Zolgensma must be used concomitantly with parenteral corticosteroids; AND
  • If the request is for a neonatal patient born prematurely, the  patient must have reached full gestational age; AND
  • The combination treatment of SMA with concomitant SMN modifying therapy/therapies will not be approved; OR
  • Zolgensma must not be used in combination with nusinersen or any SMN modifying therapy; 
    • Current authorization for any SMN modifying therapy (e.g., nusinersen) will be discontinued upon Zolgensma approval
    • If approved for Zolgensma, any subsequent request for SMN modifying therapy (e.g., nusinersen) will not be approved
      • Current authorization for nusinersen may be continued if the prescribing practitioner provides published peer-reviewed clinical research documenting a superior clinical outcome with the combination therapy of Zolgensma and nusinersen vs. monotherapy with Zolgensma.

Quantity limits

  • 1 kit per lifetime
  • Patient’s most current weight (in kg) must be provided at time of request and the dose to be administered does not exceed one kit
  • Authorization will be for up to 14 days from approval or until 2 years of age, whichever comes first

Billing for Zolgensma

Zolgensma is not covered through the MHCP fee-for-service pharmacy benefit and must be billed as a medical claim.  

Background information

Zolgensma is FDA-approved for treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.  The safety and effectiveness of repeat administration of Zolgensma have not been evaluated.  The use of Zolgensma in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated.  Use of Zolgensma in in premature neonates before reaching full term gestational age is not recommended because concomitant treatment with corticosteroids may adversely affect neurological development


MHCP Provider Call Center 651-431-2700 or 800-366-5411

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