NICE Recommends Novartis’ Zolgensma for Certain Forms of Spinal Muscular Atrophy

The UK’s National Institute for Health and Care Excellence (NICE) has recommended Novartis’ Zolgensma (onasemnogene abeparvovec) for the treatment of patients with presymptomatic 5q spinal muscular atrophy with a biallelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene in babies 12 months and under.

The recommendation for use by the National Health Service (NHS) was supported by clinical trial evidence that suggests that the treatment is effective in babies with presymptomatic spinal muscular atrophy, although there is a lack of long-term evidence, NICE said.

Spinal muscular atrophy is a rare, progressive neuromuscular condition caused by a genetic mutation which causes muscle weakness, worsening physical disability, mobility loss and respiratory dysfunction.

Zolgensma is a single-dose intravenous infusion gene therapy with a list price in Europe of 1.9 million Euro (more than $2 million). The negotiated price for NHS use was not disclosed.