Tecfidera (Dimethyl Fumarate) Approved for First-Line Treatment of Relapsing MS
FDA nod based on results from two phase III trials (Mar. 27)
The FDA has approved Tecfidera capsules (dimethyl fumarate, Biogen Idec) for the treatment of adults with relapsing forms of multiple sclerosis (MS).
The FDA approval is based on data from two phase III trials (DEFINE and CONFIRM), which enrolled more than 2,600 patients. In an ongoing extension study (ENDORSE), some patients receiving dimethyl fumarate have been followed for more than 4 years.
In the DEFINE trial, dimethyl fumarate, administered twice daily, significantly reduced the proportion of patients who relapsed by 49% (P < 0.0001), the annualized relapse rate (ARR) by 53% (P < 0.0001), and 12-week confirmed disability progression, as measured by the Expanded Disability Status Scale (EDSS), by 38% (P = 0.0050) compared with placebo at 2 years.
In the CONFIRM study, twice-daily dimethyl fumarate significantly reduced the ARR by 44% (P < 0.0001) and the proportion of patients who relapsed by 34% (P = 0.0020) compared with placebo at 2 years. While not statistically significant, dimethyl fumarate also showed a 21% reduction in 12-week confirmed disability progression.
Both studies demonstrated that dimethyl fumarate significantly reduced lesions in the brain compared with placebo, as measured by magnetic resonance imaging (MRI).
Tecfidera (dimethyl fumarate) may lower a person’s lymphocyte count. Low lymphocyte counts can increase the risk of infection, although no significant increase in infections was seen in patients taking dimethyl fumarate in clinical trials. Before starting treatment, and annually thereafter, the FDA recommends that patients’ lymphocyte counts be assessed by their health care providers.
Flushing and gastrointestinal problems (nausea, vomiting, and diarrhea) were the most common adverse reactions reported by patients treated with dimethyl fumarate in clinical studies, especially at the start of therapy. These side effects may decrease over time.