Ontario treatments

ROCHE: Evrysdi – Risdiplam



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

BIOGEN: Nusinersen – Spinraza


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



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;
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.


Cure SMA Canada Conference August 19 – 21, 20220 Montreal, Quebec Le Centre Sheraton Montréal 1201 Blvd Rene-Levesque West

‘This disease will slowly rob me of everything’: Why are Canadians living with rare diseases waiting for treatment?

COVID has proven we can quickly set up infrastructure without long-term clinical data. It’s time to do the same with rare diseases, says Durhane Wong-Rieger, president and CEO of Canadian Organization for Rare Disorders. Vanessa Hrvatin, Feb 28 2022 Sasha Kirkland, pictured with her husband, Mark and son Maks, lives with spinal muscular atrophy (SMA).…

Novartis Community Update


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