Risdiplam (Evrysdi)

CADTH recommends that Evrysdi should be reimbursed by public drug plans for the treatment of spinal muscular atrophy (SMA) in patients aged 2 months and older, if certain conditions are met. Evrysdi should only be reimbursed if the patient is under the care of a specialist with experience in the diagnosis and management of SMA, it is not used in combination with nusinersen or onasemnogene abeparvovec, and the price is reduced. Evrysdi should only be reimbursed to treat patients aged 2 months to 7 months with genetic documentation of 2 or 3 copies of the survival motor neuron 2 (SMN2) gene or non-ambulatory patients aged 8 months to 25 years with genetic documentation of 2 or 3 copies of the SMN2 gene. Patients are ineligible if they currently require permanent invasive ventilation. After 12 months of treatment, patients should be assessed to ensure clinical benefit.