Quebec Treatments

Evrysdi – Risdiplam

Available:

Effective February 2, 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.

Spinraza – Nusinersen

Link to more information

Available:

Pre-symptomatic: For the treatment of 5q spinal muscular atrophy confirmed by a genetic test showing a mutation or a biallelic deletion of the SMN1 gene among pre-symptomatic children.

Symptomatic: For the treatment of type I, II or III 5q spinal muscular atrophy confirmed by genetic test showing a mutation or a biallelic deletion of SMN1 gene. Upon the beginning of the initiation of treatment, the person must: have 2-4 copies of SMN2 gene, have the symptoms of the disease; AND not depend on PIV.

All patients: Cannot receive both nusinersen and risdiplam. Patients who have received risdiplam cannot receive nusinersen thereafter. For continued coverage, must not require PIV, not require nasogastric or gastrostomy feeding, AND must show stabilization or improvement of condition.

Zolgensma – Onasemnogene abeparvovec

Link to more information

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.