Nusinersen (Spinraza)

Key Messages

• CADTH recommends that Spinraza should not be reimbursed by public drug plans for the treatment of patients with type II and type III 5q spinal muscular atrophy (SMA) regardless of ambulatory status if initiated in patients older than 18 years of age.
• No randomized clinical trials evaluating the efficacy or safety of Spinraza in treatment-naïve adult patients with type II or type III SMA have been conducted.
• Evidence from 4 observational studies generally suggested that treatment with Spinraza may improve or maintain physical abilities; however, due to the limitations of these studies, it was not possible to conclude that the improvement or maintenance in physical function including movement or strength were a result of Spinraza.
• There is a need for treatments in adult patients with type II and type III SMA that stabilize disease progression, including the avoidance of using machines to help breathing, improve strength in the upper limbs, and improve health-related quality of life (HRQoL). However, the evidence reviewed did not show that Spinraza would meet any of these needs.