Scottish Medicines Consortium agreed access to the SMA Gene therapy Zolgensma
onasemnogene abeparvovec (Zolgensma®) is accepted for restricted use within NHSScotland.
Indication under review: treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
SMC restriction: for the treatment of-patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or
– pre-symptomatic patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene, where patients are expected to develop SMA type
In a phase III study of patients with symptomatic SMA type 1 treated with onasemnogene abeparvovec, survival was significantly better than a historical control cohort. In addition, motor milestones achieved generally exceeded the natural history of SMA type 1.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
At TreatSMA we welcome the news and will continue to work with NHSScotland and Novartis Gene Therapies to ensure suitable access is established. However, we also understand that this announcement will not affect many of the existing patients across the country and therefore we continue to work along other avenues to ensure that ALL people with SMA can have access to suitable treatment.