Earlier this week Tecfidera (dimethyl fumarate)
Earlier this week Tecfidera (dimethyl fumarate) was recommended in the EU, along with Genzyme’s rival treatment Aubagio (teriflunomide), to treat adults with relapsing-remitting MS.
Tecfidera’s US approval is significant since it is the only known drug to have demonstrated activation of the Nrf-2 pathway, which provides a way for cells to defend themselves against the inflammation caused by MS.
The FDA decision was based on data including two global Phase III studies, DEFINE and CONFIRM, in which patients received twice-daily doses of the drug.
In DEFINE, it reduced the proportion of patients who relapsed by 49%, annualised relapse rate (ARR) by 53% and 12-week confirmed disability progression – measured by the Expanded Disability Status Scale (EDSS) – by 38% compared to placebo at two years.
In CONFIRM it reduced ARR by 44% and the proportion of patients who relapsed by 34%.
In both studies the drug significantly reduced lesions in the brain compared to placebo, as measured by magnetic resonance imaging (MRI).
“We will offer the MS community a treatment with strong efficacy and a favourable safety profile in the convenience of a pill – a combination we believe will have a significant positive impact on the way people live with this chronic disease,” said Biogen chief executive George Scangos.
Injectable therapies such as Biogen’s existing Avonex and Merck Serono’s Rebif still make up the majority of the MS market but this is changing.
Competitors in pill form include Aubagio and Novartis’ Gilenya (fingolimod) – the first once-daily tablet approved to treat relapsing forms of MS.
Results from two large Phase III studies suggest Gilenya has a positive effect on relapses and MRI outcomes if used early.
MS affects an estimated 2.5 million people worldwide and is a chronic, disabling disease that attacks the central nervous system, leading at its worst to paralysis or loss of vision.