EC Expands Approval of Sanofi’s Oral MS Therapy to Include Children and Adolescents
June 21, 2021
The European Commission approved Sanofi Aubagio as the first oral MS therapy for first-line treatment of children and adolescents age 10 to 17 living with relapsing-remitting multiple sclerosis.
MS affects an estimated 2.8 million people around the world, with children and adolescents representing at least 30,000 of those impacted. Pediatric MS is a rare condition and onset follows a relapsing-remitting disease course in 98 percent of pediatric patients. Compared with adult-onset MS, pediatric patients often present with higher relapse rates and a greater lesion burden.5 Due to the earlier onset of disease, irreversible disability and secondary progression often occur at an earlier age than with adult counterparts.3 The symptoms of MS can impact all aspects of a young person’s life from physical health to social development and self-esteem.6
Aubagio was initially approved in the European Union in 2013 for the treatment of adult patients with RRMS and the EC approval for the pediatric indication provides an additional year of marketing protection in the European Union.
“Pediatric 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 pediatrics means young people with MS have a new treatment option, and importantly – one that can offer meaningful improvement in managing this serious disease.”
The Phase 3 TERIKIDS study is a multicenter, randomized, double-blind, placebo-controlled, parallel-group trial that enrolled 166 pediatric patients with relapsing-remitting forms of MS across 22 countries worldwide. The study consisted of a screening period (up to four weeks), followed by a double-blind treatment period (up to 96 weeks after randomization). An open-label TERIKIDS Phase 3 trial extension is ongoing. 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. Additionally, patients who completed the double-blind period, or had high MRI activity, were eligible to continue into the open-label extension.
The primary efficacy results and safety and tolerability data from the double-blind core study period (up to 96 weeks after randomization) were initially presented at the 2020 EAN Virtual Congress. In the study, 109 patients were randomized to teriflunomide and 57 to placebo.
The primary endpoint was not statistically significant with numerically a lower risk (-34 percent) of clinical relapse for teriflunomide vs placebo (median time: 75.3 vs 39.1 weeks). Switches from double-blind to open-label treatment due to high MRI activity were more frequent than anticipated. Switches were more frequent and earlier in the placebo group (26 percent) vs teriflunomide (14 percent). This decreased study power for the primary endpoint.
In the pre-specified sensitivity analysis of the composite endpoint of time to first clinical relapse or high MRI activity meeting study criteria to switch to open label, teriflunomide significantly reduced the time to clinical relapse or switch due to high MRI activity by 43 percent relative to placebo (median time: 72.1 vs 37.0 weeks).
In the study, teriflunomide was well tolerated and had a manageable safety profile in the pediatric population. The overall incidences of adverse events (AEs) and serious adverse events (SAEs) were similar in the teriflunomide group and the placebo group.
Photo: Erik Wallström, therapeutic area head, neurology development at Sanofi Genzyme
Author: Rare Daily Staff
Sign up for updates straight to your inbox.