Evrysdi – Risdiplam
Available:
Coverage Criteria/Therapeutic Notes
Limited use benefit (prior approval required).
For the treatment of 5q spinal muscular atrophy (SMA) if the following clinical criteria are met.
Initial Coverage Criteria (12 months):
– Genetic documentation of 5q SMA homozygous gene deletion or compound heterozygote; AND
– Patient is under the care of a specialist with experience in the diagnosis and management of SMA; AND
– Patient is symptomatic; AND
– is aged between 2 months and 7 months (inclusive); AND has genetic documentation of two or three copies of the SMN2 gene
OR
– is aged 8 months up to 25 years; AND is non-ambulatory; AND has genetic documentation of two or three copies of the SMN2 gene
AND
– Patient does not (or did not) require permanent invasive ventilation* at treatment initiation.
*Permanent invasive ventilation defined as the need for a tracheostomy or requirement of 16 hours or more of non-invasive ventilation (e.g., BIPAP) per day or intubation for more than 21 consecutive days in the absence of, or following the, resolution, of an acute reversible event.
A baseline assessment using an age-appropriate scale must be completed prior to EVRYSDI administration using ONE of the following baseline symptom assessment scores:
– Hammersmith Infant Neurological Examination (HINE) Section 2 score
– Children¿s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
– Hammersmith Functional Motor Scale-Expanded (HFMSE) score
Renewal Coverage Criteria (12 months):
Treatment with Evrysdi (risdiplam) should be DISCONTINUED if any of the following occur:
– there is no demonstrated achievement in, or maintenance of, motor milestone function (as assessed using an age-appropriate measurement) after treatment initiation in patients aged between 2 months and 2 years at the time of treatment initiation
– there is no demonstrated maintenance of motor function (as assessed using an age-appropriate measurement) after treatment initiation in patients aged between 2 years and 25 years at the time of treatment initiation
– permanent invasive ventilation is required
If a patient has not demonstrated maintenance of motor function based on the current assessment, then the results should be validated with a confirmatory assessment conducted no longer than 12 weeks later.
An assessment must be completed using ONE of the following baseline symptom assessment scores. This should be the same scoring system submitted for the baseline assessment.
– Hammersmith Infant Neurological Examination (HINE) Section 2 score
– Children¿s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
– Hammersmith Functional Motor Scale-Expanded (HFMSE) score
Combination use of Evrysdi (risdiplam) and Spinraza (nusinersen) or Zolgensma (onasemnogene abeparvovec) will not be funded.