Onasemnogene abeparvovec (Zolgensma®) is accepted for restricted use within NHSScotland.
Accepted for 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.
Source:
Scottish Medicines Consortium
SPS commentary:
This treatment has also been accepted by NHSEngland with reported list price of £1.79m per dose will be available to patients at a price that is fair to taxpayers after a deal struck by NHSE. This will allow up to 80 patients a year to benefit from single IV infusion therapy containing a replica of missing gene SMN1.