Prince Edward
Island Treatments

Evrysdi – Risdiplam

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Available:

EVRYSDIⓇ (risdiplam) has been added to the PEI High Cost Drug Program formulary effective March 27th, 2023 For patients diagnosed with 5q Spinal Muscular Atrophy (SMA) under the care of a specialist with experience in the diagnosis and management of SMA, if the following clinical criteria are met: • Genetic documentation of 5q SMA homozygous gene deletion or compound heterozygote, AND • Patients who: o are symptomatic and have genetic documentation of two or three copies of the SMN2 gene, AND o aged between 2 months and 7 months (inclusive), OR o aged 8 months up to 25 years and are non-ambulatory • Patient is not currently requiring permanent invasive ventilation*, AND • A baseline assessment using an age-appropriate scale (the Hammersmith Infant Neurological Examination [HINE] Section 2, Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders [CHOP INTEND], or Hammersmith Functional Motor Scale-Expanded [HFMSE]) must be completed prior to initiation of risdiplam treatment. • For continued coverage, the patient must meet the following criteria: There is demonstrated achievement or maintenance of motor milestone function (as assessed using age-appropriate scales: the [HINE] Section 2, CHOP INTEND, or HFMSE) after treatment initiation in patients aged between 2 months and 2 years at the time of treatment initiation; OR o There is demonstrated maintenance of motor milestone function (as assessed using age-appropriate scales: the HINE Section 2, CHOP INTEND, or HFMSE) after treatment initiation in patients aged between 2 years and 25 years at the time of treatment initiation; AND o Patient does not require permanent invasive ventilation*. The decision to discontinue reimbursement should be based on 2 assessments separated by no longer than a 12-week interval. Claim Notes: • Approval: 12 months

The specific criteria for reimbursement are in line with recommendations made by CADTH in 2021.

*Permanent invasive ventilation is defined as the use of tracheostomy and a ventilator due to progression of SMA that is not due to an identifiable and reversible cause.

Spinraza – Nusinersen

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Available:

Pediatric:

Initiation Criteria
1. Pre-symptomatic patients with 2 or 3 copies of the SMN2 gene,
OR
2. Had the disease for <6 months, 2 copies of SMN2, and symptom onset after the first week after birth and on, or before seven months of age.
OR
3. Patients under the age of 18 with symptom onset > 6 months of age, and never achieved the ability to walk independently. †

Stopping Criteria
1. For those pre-symptomatic at initiation: no improvement/maintenance on HINE-2, CHOP INTEND, or HFMSE, OR
2. For those symptomatic at initiation: no maintenance in HINE-2, CHOP INTENT, or HFMSE, OR
3. Permanent invasive ventilation required

Adult:

“Other patients with SMA type 2 or 3 who are over the age of 18 may be considered on a case-by-case basis.”

Zolgensma – Onasemnogene abeparvovec

Available:

No information available at this time.