FDA Approves Third Oral Agent for MS

Susan Jeffrey

March 27, 2013

The US Food and Drug Administration (FDA) has approved dimethyl fumarate (Tecfidera, Biogen Idec) for the treatment of relapsing-remitting multiple sclerosis (MS).

The approval brings to 3 the number of oral agents now approved for this indication. Fingolimod (Gilenya, Novartis) was the first, in September 2010, followed by teriflunomide (Aubagio, Genzyme/Sanofi) earlier this year.

"No drug provides a cure for multiple sclerosis, so it is important to have a variety of treatment options available for patients," said Russell Katz, MD, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research, in a statement.

The drug acts by reducing lymphocytes, which can raise the risk for infection, so the FDA is recommending monitoring before the start of treatment and then annually thereafter, the statement notes.

Flushing and stomach problems, including nausea, vomiting, and diarrhea, were the most common adverse reactions reported by patients receiving the drug in clinical trials, particularly at the start of therapy. These adverse effects may decrease over time.

The application is based on findings from the Determination of the Efficacy and safety of oral Fumarate IN rElapsing-remitting MS (DEFINE) trial and the Comparator and an Oral Fumarate in RRMS (CONFIRM) trial, both published in the September 20 issue of the New England Journal of Medicine. The trials were funded by Biogen Idec.

Full results of the 2 phase 3 trials evaluating the effect of oral dimethyl fumarate in patients with MS showed that the investigational agent reduced relapse rates, disease activity on imaging, and, in 1 trial, progression of disability.

In October 2012, the company announced that FDA had extended review of dimethyl fumarate for an additional 3 months to allow more time to review the application, although without asking for additional studies.

Biogen Idec received a new patent for the 480-mg dosing regimen of dimethyl fumarate earlier this month that will protect their exclusivity with the drug until 2028.

On March 22, the European Medicines Agency's Committee for Medicinal Products for Human Use recommended marketing authorization for both dimethyl fumarate and teriflunomide in the European Union as first-line oral agents in the treatment of relapsing-remitting MS.