British Columbia treatments
ROCHE: Evrysdi – Risdiplam
https://news.gov.bc.ca/releases/2022HLTH0055-000225
Available:
Effective February 16, 2022 – Available through Exceptional Expensive Drugs for Rare Disease Process – no criteria available
BIOGEN: Nusinersen – Spinraza
https://archive.news.gov.bc.ca/releases/news_releases_2017-2021/2019HLTH0164-002427.pdf
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
British Columbia covers only pediatric patients up to the age of 12.
NOVARTIS: Onasemnogene abeparvovec – Zolgensma
Available
EDRDs considered for exceptional case-by-case funding
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