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

https://www2.gov.bc.ca/gov/content/health/health-drug-coverage/pharmacare-for-bc-residents/what-we-cover/exceptional-funding-edrd

Available

EDRDs considered for exceptional case-by-case funding