Evrysdi – Risdiplam
Effective March 28, 2022 – Initiation Criteria:
For the treatment of spinal muscular atrophy (SMA) in patients meeting all the following
1. Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation,
or compound heterozygote; AND
2. Has genetic documentation of 2 or 3 copies of the SMN2 gene; AND
3. Patient is symptomatic; AND
4. Aged between 2 months to 7 months (inclusive) OR Is non-ambulatory and aged 8
months to 25 years (inclusive); AND
5. Patient is under the care of a specialist experienced in the diagnosis and management
1. Patients who meet all the initiation criteria but who are ambulatory may be
considered on a case-by-case basis.
2. Patients who are pre-symptomatic or asymptomatic may be considered on a caseby-case basis.
3. Age-appropriate measurements include the Hammersmith Infant Neurological
Examination (HINE) Section 2, Children’s Hospital of Philadelphia Infant Test of
Neuromuscular Disorders (CHOP INTEND), Hammersmith Functional Motor ScaleExpanded (HFMSE), and the Revised Upper Limb Module (RULM) Scores. You
may submit the evaluations from more than one age-appropriate test to support your
4. Requests to switch from nusinersen to risdiplam and vice versa will be considered
on a case-by-case basis for patients who meet inclusion criteria and who have not
met discontinuation criteria on their current SMA treatment.
• Patient with SMA type 4 will not be funded.
• Patient with more than four (4) SMN2 gene copies will not be funded.
• Patient with permanent invasive ventilation (PIV) will not be funded.
• Risdiplam is not funded in patients as combination therapy with nusinersen.
• Risdiplam is not funded in patients who have received onasemnogene abeparvovec.
Renewal of funding will be considered for patients who do not meet any of the exclusion criteria AND who have not demonstrated any of the stopping/discontinuation criteria while on therapy.
Funded Dose: Age and weight appropriate doses of 0.2 mg/kg to 0.25 mg/kg up to a maximum dose of 5 mg daily.
Approval Duration of Initials and Renewals: 12 months