Itvisma®

Drug - Itvisma® (onasemnogene abeparvovec-brve) [Novartis Pharmaceutical Corporation]

September 2026

Therapeutic area - Spinal Muscular Atrophy

Initial approval criteria

  • Patient must be ≥ 2 years of age; AND
  • Patients has a diagnosis of spinal muscular atrophy (SMA); AND
  • Patient has a diagnosis of 5q SMA confirmed by either bi-allelic deletion or dysfunctional point mutation of the survival motor neuron 1 (SMN1) gene; AND
  • Patient must NOT have advanced disease (e.g., complete limb paralysis, permanent ventilation support); AND
    • Patient is treatment-naïve for any SMN-targeting agents for SMA (e.g., nusinersen [Spinraza], risdiplam [Evrysdi]); OR
    • Patient has received prior SMN-targeting agents for SMA (e.g., Spinraza, Evrysdi) AND has one or more of the following:
      • Able to sit independently, but has never had the ability to walk independently; OR
      • Has up to 4 copies of SMN2 gene; OR
      • Achieved and subsequently lost the ability to walk independently; AND
  • Will only be administered intrathecally using a lumbar puncture by healthcare professionals (e.g., interventional radiologist or neurologist) experienced in performing lumbar punctures; AND
  • Patient is clinically stable in their overall baseline health status (e.g., hydration and nutritional status, respiratory status) prior to administration; AND
  • Patient does NOT have an active infection, including clinically important localized infections; AND
  • Patient must have a baseline anti-adeno-associated virus serotype 9 (anti-AAV9) antibody titer of ≤ 1:50 measured by ELISA; AND
  • Baseline liver function will be assessed prior to initiating therapy and will continue to be monitored for at least 3 months after therapy and as clinically indicated; AND
  • Baseline platelet counts will be assessed prior to initiating therapy and will continue to be monitored on a regular basis (e.g., at least weekly for the first month and as clinically indicated until platelet counts return to baseline); AND
  • Patient is up to date with all vaccinations in accordance with current vaccination guidelines, prior to initiating therapy; AND
  • Used concomitantly with systemic corticosteroids; AND
  • Patient will be considered for neurologic or cardiac evaluation based on clinical presentation; AND
  • Patient must NOT have previously received treatment with SMA gene therapy (e.g., onasemnogene abeparvovec-xioi [Zolgensma]); AND
  • Will NOT be used in combination with other SMN-targeting agents for SMA (e.g., Spinraza, Evrysdi, Zolgensma); AND
  • Females of reproductive potential should have a negative pregnancy test prior to receiving Itvisma and should use effective contraception (methods that result in <1% pregnancy rates) and should refrain from egg donation for 6 months following Itvisma administration; AND
  • Male patients with female partners of reproductive potential should use a barrier method of contraception and should refrain from sperm donation for 3 months following Itvisma administration.

Quantity limits

  • One treatment course and may not be renewed

Billing for Itvisma

Itvisma must be billed as a professional claim.

Questions

Provider Call Center: 844-575-7887