Evrysdi™

Drug - Evrysdi™ (risdiplam oral solution) [Genentech Inc.]

March 2021

Therapeutic area - Spinal Muscular Atrophy (SMA)

Initial approval criteria

• Patient ≥ 2 months of age AND
• Patient has a diagnosis of 5q-autosomal recessive spinal muscular atrophy (SMA) confirmed by either homozygous deletion of the SMN1 gene or dysfunctional mutation of the SMN1 gene AND
• Patient must have 1 of the following phenotypes:
  • SMA type 1 with symptomatic disease (e.g., impaired motor function and/or delayed motor milestones) OR
  • SMA type 2 with symptomatic disease (e.g., impaired motor function and/or delayed motor milestones) OR
  • SMA type 3 with symptomatic disease (e.g., impaired motor function and/or delayed motor milestones) AND
• Risdiplam must NOT be used concomitantly with nusinersen (Spinraza®) or onasemnogene abeparvovec-xioi (Zolgensma®) AND
• Patient must NOT have previously received onasemnogene abeparvovec-xioi (Zolgensma) AND
• Patient has baseline documentation of ≥ 1 of the following:
  • Motor function/milestones, including but NOT limited to, the following validated scales: Hammersmith Infant Neurologic Exam (HINE), Hammersmith Functional Motor Scale Expanded (HFMSE), Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), 6-minute walk test (6MWT), upper limb module (ULM), etc. 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) AND
• Prescriber attests that the patient or patient’s caregiver has been counseled on the proper use and storage of medication (e.g., what to do if missing or vomiting a dose; how to use the reusable oral syringes to transfer dose from bottle, etc.) AND
• Prescriber attests that the patient or patient’s caregiver has been instructed on all the steps needed to ensure a successful delivery of medication from the specialty pharmacy

Renewal criteria

• Patient continues to meet the above initial criteria AND
• Patient is absent of unacceptable toxicity or treatment related adverse event from the drug AND
• Patient has experienced a clinically meaningful response to treatment as demonstrated by ≥ 1 of the following:
  • Stability or improvement in net motor function/milestones, including but not limited to, the following validated scales: Hammersmith Infant Neurologic Exam (HINE), Hammersmith Functional Motor Scale Expanded (HFMSE), Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), Bayley Scales of Infant and Toddler development Third Edition (BSID-III), 6-minute walk test (6MWT), upper limb module (ULM), etc. OR
  • Stability or improvement in respiratory function tests [e.g., forced vital capacity (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

Quantity limits

• 2 months to less than 2 years of age:  0.2 mg/kg/day
• 2 years of age and older weighing less than 20 kg:  0.25 mg/kg/day
• 2 years of age and older weighing 20 kg or more:  5mg/day
• Patient’s weight (in kg) must be provided at time of request
• Requested total amount (in mg) for each fill and the corresponding requested number of bottles for each fill that will minimize waste
• Requested number of refills must be clearly stated on the prior authorization request form
• Refill-too-soon requests will not be considered for lost, stolen, or damaged medication while in transit, vomited medication, or spoiled medication due to improper storage conditions

Questions?

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