Evrysdi – Risdiplam
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.