There are now two approved treatment options for children and young people with MS in some parts of the world: teriflunomide (Aubagio) and fingolimod (Gilenya).
MS is estimated to affect at least 30,000 children and adolescents globally, according to the latest data in the Atlas of MS. Many of the disease modifying therapies (DMTs) prescribed for adults with MS are also prescribed for children. This is described as ‘off-label’ prescribing, because the treatments have not been specifically licensed for children.
The first treatment approved for children with MS
Until recently, only one DMT – fingolimod – had regulatory approval for children with MS. In 2018, the Food and Drug Administration (FDA) in the United States approved fingolimod (Gilenya) as a treatment for children and young people aged 10 years and older with relapsing MS.
Teriflunomide: why was it approved by one regulator and rejected by another?
In June 2021, the European Medicines Agency (EMA) approved the drug teriflunomide for children with MS aged 10 and older, whereas the FDA rejected the application. These different decisions relate to the type of evidence the two regulatory agencies require in order to show that a drug is effective.
The EMA will accept positive outcomes that are shown on an MRI as evidence (e.g. a reduced number of lesions), but the FDA will only approve treatments that show positive clinical outcomes, such as a delay in the time to the first MS attack.
What the clinical trial showed
The Phase III clinical trial that this data was based on was called TERIKIDS. It compared outcomes in children with MS who were taking teriflunomide to those taking a placebo treatment. The data showed positive MRI outcomes in the teriflunomide arm of the trial, but was not able to demonstrate positive clinical outcomes with statistical significance.
This is because many participants were removed from the study before it ended. Participants were monitored throughout the trial, and anyone who showed a lot of MRI activity (which would signal active disease) was removed from the trial so they could be given a known treatment. This is because it would have been unethical to keep them in the trial in case they were in the placebo arm of it.
Whilst teriflunomide for children and young people with MS does not have regulatory approval globally, this is an encouraging step towards providing a greater choice of approved DMTs for this younger group of people with MS.
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