New product: Zolgensma (onasemnogene abeparvovec) 2 x 1013 vector genomes/mL solution for infusion
This gene therapy is licensed for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and ≤3 copies of SMN2 gene.
Source:
electronic Medicines compendium
SPS commentary:
In the U.S, this treatment is reported to cost $2.1 million. It has been scheduled into NICE work programme and is due to be discussed by the Highly Specialised Technology committee on 8 October 2020.