Ontario treatments

ROCHE: Evrysdi – Risdiplam

https://www.health.gov.on.ca/en/pro/programs/drugs/odbf/odbf_except_access.aspx

Available:

Effective March 28, 2022 – Available through the Exceptional Drug Status Program; criteria isn’t yet public

BIOGEN: Nusinersen – Spinraza

Available:

Initiation Criteria
1. Pre-symptomatic patients with two or three copies of the SMN2 gene, OR

2. Had the disease for <6 months, two 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 on HINE-2, CHOP INTEND, or HFMSE, OR

2. For those symptomatic at initiation: no improvement in HINE-2, CHOP INTENT, or HFMSE, OR

3. Permanent invasive ventilation required

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

NOVARTIS: Onasemnogene abeparvovec – Zolgensma

https://health.gov.on.ca/en/pro/programs/drugs/docs/frequently_requested_drugs.pdf

Available

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