The European Commission has approved Sanofi’s oral multiple sclerosis (MS) therapy Aubagio for the first-line treatment of paediatric patients aged ten to 17 years living with relapsing/remitting MS (RRMS).
Although paediatric MS is a rare condition, compared with adult-onset MS, paediatric patients often present with higher relapse rates and a greater lesion burden.
“Paediatric multiple sclerosis remains an area of significant unmet medical need,” said Erik Wallström, therapeutic area head, neurology development at Sanofi Genzyme.
“The European approval of Aubagio in paediatrics means young people with MS have a new treatment option, and importantly – one that can offer meaningful improvement in managing this serious disease,” he added.
The approval of Aubagio (teriflunomide) in this patient population is based on data from the Phase III TERIKIDS study, which enrolled 166 paediatric patients with RRMS across 22 countries worldwide.
The primary endpoint was time to first confirmed clinical relapse, with prespecified sensitivity analysis including time to high magnetic resonance imaging (MRI) activity as relapse equivalent. Patients who completed the double-blind period, or who had high MRI activity, were eligible to continue into the open-label extension.
Although the primary endpoint was not statistically significant, there was a numerically lower risk (-34%) for Aubagio versus placebo. According to Sanofi, switches from double-blind to open-label treatment due to high MRI activity occurred more frequently ‘than anticipated’.
In addition, switches were more frequent and earlier in the placebo group versus Aubagio, which decreased study power for the primary endpoint, according to Sanofi.
In a prespecified sensitivity analysis of the composite endpoint of time to first clinical relapse or high MRI activity meeting the study criteria to switch to open-label, Aubagio significantly reduced the time to clinical relapse or switch due to high MRI activity by 43% compared to placebo.
Secondary endpoints also showed that Aubagio significantly reduced the number of T1 gadolinium (Gd) enhancing lesions per MRI scan, as well as the number of new and enlarging T2 lesions per MRI scan.