Evrysdi (risdiplam) — CareFirst (Caremark)

Initial criteria

• Member has type 1, type 2, or type 3 SMA
• Genetic documentation of 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote
• Member is not dependent on invasive ventilation or tracheostomy
• Member is not dependent on non-invasive ventilation beyond naps and nighttime sleep
• Member has not previously received gene replacement therapy for SMA (e.g., Zolgensma), OR if member has previously received gene replacement therapy for SMA (e.g., Zolgensma) the member has experienced a worsening in clinical status as demonstrated by a decline from baseline or highest score achieved on one of the following assessments (based on age and motor ability): HINE-2 (decline of ≥2 points on kicking and 1 point on any other milestone excluding voluntary grasp); HFMSE (decline ≥3 points); CHOP-INTEND (decline ≥4 points); MFM32 (decline from baseline); BSID-III (inability to sit without support for more than 5 seconds on item 22)
• Member will not use Evrysdi and Spinraza concomitantly
• Daily dose does not exceed: <2 months: 0.15 mg/kg; 2 months–<2 years: 0.2 mg/kg; ≥2 years and <20 kg: 0.25 mg/kg; ≥2 years and ≥20 kg: 5 mg
• Medication must be prescribed by or in consultation with a physician who specializes in treatment of spinal muscular atrophy

Reauthorization criteria

• Member has type 1, type 2, or type 3 SMA
• Member is not dependent on invasive ventilation or tracheostomy
• Member is not dependent on non-invasive ventilation beyond naps and nighttime sleep
• Submission of medical records (less than 1 month prior to continuation request) documenting positive clinical response from pretreatment baseline to Evrysdi therapy as evidenced by at least one of the following:
• HINE-2: improvement or maintenance of ≥2-point (or maximal score) increase in ability to kick, OR ≥1-point (or maximal score) increase in any other milestone excluding voluntary grasp, AND net positive improvement in motor milestones or achievement/maintenance of new milestones (e.g., sit or stand unassisted, walk)
• HFMSE: improvement or maintenance of ≥3-point increase in score, OR achievement/maintenance of new motor milestone from baseline
• CHOP-INTEND: improvement or maintenance of ≥4-point increase in score, OR achievement/maintenance of new motor milestone from baseline
• MFM32: increase from baseline correlated with clinically significant functional improvement
• BSID-III: ability to sit without support for at least 5 seconds after 12 months of treatment
• If prescribed due to clinical worsening after gene replacement therapy (e.g., Zolgensma): documentation of stabilization or improvement in clinical status with Evrysdi therapy
• Member will not use Evrysdi and Spinraza concomitantly
• Daily dose does not exceed: <2 months: 0.15 mg/kg; 2 months–<2 years: 0.2 mg/kg; ≥2 years and <20 kg: 0.25 mg/kg; ≥2 years and ≥20 kg: 5 mg

Approval duration

12 months