Temporary Change to Zolgensma® Treatment Eligibility Emphasises Need for Newborn Screening for SMA
SMA UK, Muscular Dystrophy UK and TreatSMA recently issued an update related to a decision by NHS England to temporarily change eligibility for NHS-funded treatment Zolgensma in England, pausing treatment in children over 12 months old due to a small number of adverse events in this cohort of patients. Recently, a small number of children treated in England, particularly those who are older and weighing more than 13.5 kg, have been noted to have suspected significant adverse drug reactions affecting the liver following the administration of Zolgensma®.
This development underscores the need for newborn screening for SMA to maximise the chance that babies with the condition get access to treatment at the time when it can deliver maximum benefit (ideally prior to symptom onset when irreversible neuron damage will have occurred) and with the minimum risk of harm (for example, via adverse reactions which may become more likely with increased age/weight at the time of treatment).
The Alliance is certain that this will be taken into consideration by UK NSC as they progress their assessment of SMA for newborn screening. We also hope that it adds increased impetus to the need for a rapid decision on newborn screening for SMA.
The temporary change will mean that, “NHS funded treatment with Zolgensma® in England should be temporarily paused in children older than 12 completed months as per NICE guidance.” The same approach is expected to be adopted across the UK.
Over 90 children have now received Zolgensma® for SMA in England. Recently, “…a small number of these, particularly those who are older and weighing more than 13.5 kg, have been noted to have suspected significant adverse drug reactions affecting the liver following the administration of Zolgensma®. Liver injury is a known adverse event associated with the therapy and contained in the product’s label. These children are being cared for and monitored by their local clinical teams, the infusion centres and other specialists as required.”
Further details and a series of questions and answers on this topic can be found in the patient organisation update here.