Traitements québécois

ROCHE: Evrysdi – Risdiplam

https://www.ramq.gouv.qc.ca/sites/default/files/documents/liste_med_2022-02-02_fr.pdf

Available:

Effective February 1, 2022 –
for treatment of 5q spinal muscular atrophy confirmed by a genetic test showing a biallelic mutation or deletion of the SMN1 gene.
Upon initiation of treatment, the person must:
• be aged 2 months and older; and
• show two, three or four copies of the SMN2 gene; and
• show symptoms of the disease; and
• do not depend on permanent ventilation. Permanent ventilation is defined by the use of respiratory assistance (invasive or non-invasive) for 16 hours or more per day, during more than 21 consecutive days, except where it is related to a reversible acute episode; and
• have discontinued treatment with nusinersen, if applicable; and
• not have been treated with onasemnogene abeparvovec.

Authorizations are given for a maximum dose of 5 mg per day. The maximum duration of each authorization is 12 months.
Persons treated with risdiplam cannot be treated with nusinersen afterwards.

When requesting continuation of treatment, the physician must provide information making it possible to establish the absence of significant deterioration of the motor functions, namely:
• the absence of permanent ventilation; and
• the absence of exclusive nasogastric or gastrostomy feeding; and
• a stabilization or improvement of the medical condition.

BIOGEN: Nusinersen – Spinraza

https://www.ramq.gouv.qc.ca/sites/default/files/documents/liste_med_etab_2022-04-13.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

Pre-symptomatic: “For treatment of 5q spinal muscular atrophy confirmed by a genetic test showing a mutation or a deletion of the SMN1 gene in presymptomatic people… Person must have 2-4 copies of the SMN2 gene; and not have been treated with risdiplam or onasemnogene abeparvovec.”

Symptomatic: “For treatment of 5q spinal muscular atrophy confirmed by a genetic test showing a mutation or biallelic deletion of the SMN1 gene…Person must have 2-4 copies of the SMN2 gene; and show symptoms of the disease; and not depend on permanent ventilation; and not have been treated with risdiplam or onasemnogene abeparvovec.”

NOVARTIS: Onasemnogene abeparvovec – Zolgensma

https://www.ramq.gouv.qc.ca/sites/default/files/documents/liste_med_etab_2022-02-02.pdf

Available

For the treatment of 5q spinal muscular atrophy confirmed by a genetic test showing a mutation or a biallelic deletion of the SMN1 gene. At the time of the infusion, the child must:
• be less than 6 months old; AND
• have two or three copies of the SMN2 gene; AND
• not rely exclusively on nasogastric or gastrostomy feeding; AND
• not rely on permanent ventilation. Permanent ventilation is defined by the use of a
ventilation support (invasive or non-invasive) for 16 hours or more per day, for more than 21 days
consecutive, unless it is related to an acute reversible episode.
Authorization is given for a maximum single dose of 1.1 x 1014 vg/kg.
If the child is treated with nusinersen, nusinersen must be discontinued for the child to be eligible treatment with onasemnogene abeparvovec, and not resumed thereafter.
It is possible that children with spinal muscular atrophy could not have received the medication before the age of 6 months. In this case, multidisciplinary committees, such as the suitability committees of the centers hospitals, may consider and authorize requests for children aged 6 months or over, based on clinical criteria, as long as they meet the aforementioned requirements, except for the one concerning age.

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