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Evrysdi — Medical Mutual

Spinal Muscular Atrophy (SMA)

Initial criteria

  • Patient must not have previously received treatment with SMA gene therapy (i.e., onasemnogene abeparvovec-xioi); AND
  • Patient will not use in combination with other agents for SMA (e.g., onasemnogene abeparvovec, nusinersen, etc.); AND
  • Patient does not have impaired hepatic function as defined by elevated laboratory values; AND
  • Patient retains meaningful voluntary motor function (e.g. manipulate objects using upper extremities, ambulate, etc.); AND
  • Patient must have a diagnosis of 5q spinal muscular atrophy confirmed by either homozygous deletion of the SMN1 gene or dysfunctional mutation of the SMN1 gene; AND
  • Patient must have one of the following SMA phenotypes: SMA I or II confirmed by genetic testing [documentation required]; OR SMA III confirmed by genetic testing [documentation required] with symptomatic disease (i.e., impaired motor function and/or delayed motor milestones); AND
  • Baseline documentation of one or more of the following: Motor function/milestones using validated scales (HINE, HFMSE, CHOP INTEND, 6MWT, ULM, MFM32, RULM, etc.); OR Respiratory function tests (e.g., FVC); OR Exacerbations necessitating hospitalization and/or antibiotic therapy for respiratory infection in the preceding year/timeframe; OR Patient weight (for patients without a gastrostomy tube)

Reauthorization criteria

  • Patient must not have previously received treatment with SMA gene therapy (i.e., onasemnogene abeparvovec-xioi); AND
  • Absence of unacceptable toxicity which would preclude safe administration of the drug (e.g., significant hepatic impairment); AND
  • Patient has responded to therapy compared to pretreatment baseline in one or more of the following: Stability or improvement in net motor function/milestones using validated scales (HINE, HFMSE, CHOP INTEND, 6MWT, ULM, MFM32, RULM, etc.); OR Stability or improvement in respiratory function tests (e.g., FVC); OR Reduction in exacerbations necessitating hospitalization and/or antibiotic therapy for respiratory infection in the preceding year/timeframe; OR Stable or increased patient weight (for patients without a gastrostomy tube); OR Slowed rate of decline in the aforementioned measures

Approval duration

initial 6 months, renewal 12 months