Ontario Treatments

Evrysdi – Risdiplam

Available:

Effective March 28, 2022 – Initiation Criteria:
For the treatment of spinal muscular atrophy (SMA) in patients meeting all the following
criteria:
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
of SMA.

Notes:
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
renewal request.
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.

Exclusion criteria:
• 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 criteria:
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

Spinraza – Nusinersen

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 who do not meet the expanded funding criteria may be considered in exceptional cases.”

Zolgensma – Onasemnogene abeparvovec

Link to more information

Available:

• Diagnosis of SMA is confirmed by genetic documentation of 5q spinal muscular atrophy with biallelic mutations in the survival motor neuron 1 (SMN1) gene; AND
• Patient is 180 days of age or younger at the time that the treatment is administered; AND
• Patient is pre-symptomatic or symptomatic with one to three copies of the survival motor neuron 2 (SMN2) gene; AND
• Patient does not require permanent ventilatory support (invasive or non-invasive)* or a permanent feeding tube; AND
• Patient must be under the care of a specialist with experience in the diagnosis and management of spinal muscular atrophy.

Exclusion Criteria:
• 1.Reimbursement is limited to one lifetime administration of onasemnogene abeparvovec. Patients who have received a prior dose of onasemnogene abeparvovec accessed by any mechanism (e.g. private insurance plan, clinical trial, compassionate access) will not be funded.
• Patients with 4 (four) or more copies of SMN2 gene will not be funded.

Notes:
• Patients must test for the presence of anti-AAV9 antibodies and provide the report of antibody titers. It should be noted that patients with titers above 1:50 will not be funded. Repeat testing results will be considered for newborns suspected of having acquired interim antibodies from maternal transmission.
• No further treatment with nusinersen or other medications indicated for the treatment of SMA will be considered after the patient has received a dose of onasemnogene abeparvovec.