NICE provides guidance for Zolgensma use in presymptomatic babies with SMA

The UK’s National Institute for Health and Care Excellence (NICE) is recommending the use of Zolgensma (onasemnogene abeparvovec) as an option in babies with presymptomatic 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene, Swiss Pharma giant Novartis (NOVN: VX) revealed today.

Zolgensma, which has a list price of £1.8 million ($2.6 million) per single dose, was made available on the National Health Service (NHS) following a landmark deal struck with Novartis Gene Therapies in March 2021. The first baby was treated with the drug in June that year.

This recommendation will allow routine access to onasemnogene abeparvovec, which is designed to address the genetic root cause of SMA to help halt disease progression, before the onset of SMA symptoms. Treatment at this early presymptomatic stage of the disease is critical to ensure babies with SMA have the best chance for optimal outcomes.