Ontario Treatments

Ontario provides coverage for the three available SMA treatments through their Exceptional Access Program (EAP). Please see below for coverage details of each of the available SMA treatments. Please discuss your eligibility with your health-care provider.

Evrysdi – Risdiplam

  • Coverage criteria can be found here by searching for ‘Evrysdi’ in the PDF document. For convenience, full coverage criteria is posted below.

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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
  1. Has genetic documentation of 2 or 3 copies of the SMN2 gene; AND
  2. Patient is symptomatic; AND
  3. Aged between 2 months to 7 months (inclusive) OR Is non-ambulatory and aged 8 months to 25 years (inclusive); AND
  1. Patient is under the care of a specialist experienced in the diagnosis and management of SMA.

 

Discontinuation Criteria:

Treatment should be discontinued upon meeting any of the following circumstances;

  • 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. It should be noted that the decision to discontinue reimbursement should be based on 2 assessments separated by no longer than a 12-week interval, with the first evaluation taken close to (i.e. within 3 months) of the date of renewal of funding. The second assessment is only required for patients who demonstrated a decline in motor milestones/motor function at the time of the first evaluation.

 

Notes:

  1. Patients who meet all the initiation criteria but who are ambulatory may be considered on a case-by-case basis.
  1. Patients who are pre-symptomatic or asymptomatic may be considered on a case-by-case basis.
  1. 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 Scale-Expanded (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.
  1. 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

  • Coverage criteria can be found here by searching for ‘Zolgensma’ in the PDF document. For convenience, full coverage criteria is posted below.

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For the treatment of spinal muscular atrophy (SMA) in individuals meeting all the

following criteria:

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

*Permanent ventilatory support is defined as the need for a tracheostomy or requirement of 16 hours or more of respiratory assistance per day (via non-invasive ventilatory support) for 14 or more consecutive days in the absence of an acute reversible illness excluding perioperative ventilation.

 

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.
  1. Patients with 4 (four) or more copies of SMN2 gene will not be funded.

 

Notes:

  1. 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.
  1. 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.

 

Dosage recommended: A maximum single lifetime dose of 1.1 x 1014 vector genomes (vg)/kg

 

Renewal of therapy is not considered.

The information on this page was last updated in May 2024.