The US Food and Drug Administration (FDA) has approved Biogen Idec's Tecfidera (dimethyl fumarate), a new first-line oral treatment for people with relapsing forms of multiple sclerosis (MS). Biogen Idec will make this oral capsule available to people living with MS in the United States in the coming days.

Tecfidera has been clinically proven to significantly reduce important measures of disease activity, including relapses and development of brain lesions, as well as to slow disability progression over time, while demonstrating a favourable safety and tolerability profile.

“With the FDA approval of Tecfidera, 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 George A. Scangos, chief executive officer of Biogen Idec. “Biogen Idec is committed to delivering innovative treatments and setting new standards for the next generation of medicines. We believe Tecfidera  will raise expectations for what people living with MS can achieve with their therapy.”

The FDA approval of Tecfidera is based on data from a robust clinical development programme that included DEFINE and CONFIRM, two global Phase 3 studies that enrolled more than 2,600 patients. In the ongoing extension study, ENDORSE, some patients receiving Tecfidera have been followed for more than four years.

In DEFINE, Tecfidera, administered twice daily, significantly reduced the proportion of patients who relapsed by 49 per cent (p<0.0001), the annualized relapse rate (ARR) by 53 per cent (p<0.0001), and 12-week confirmed disability progression, as measured by the Expanded Disability Status Scale (EDSS), by 38 per cent (p=0.0050) compared to placebo at two years. In CONFIRM, twice-daily Tecfidera significantly reduced ARR by 44 per cent (p<0.0001) and the proportion of patients who relapsed by 34 per cent (p=0.0020) compared to placebo at two years. While not statistically significant, Tecfidera showed a 21 per cent reduction in 12-week confirmed disability progression in CONFIRM. Both studies also showed that Tecfidera significantly reduced lesions in the brain compared to placebo, as measured by magnetic resonance imaging (MRI).

“In clinical trials, patients treated with dimethyl fumarate had less disease activity when compared to patients on placebo – whether they were in the early stages of MS or had more established disease,” said Robert Fox, medical director of the Mellen Center for Multiple Sclerosis at Cleveland Clinic, lead investigator of the CONFIRM study, and a paid advisor for Biogen Idec for projects not related to Tecfidera clinical development. “With the efficacy, safety and tolerability measures seen in CONFIRM, this drug provides physicians with an important additional treatment option for their patients across the MS spectrum.”

The most common side effects associated with Tecfidera are flushing and gastrointestinal (GI) events (i.e., diarrhoea, nausea and abdominal pain). In clinical studies, flushing symptoms usually began soon after initiating treatment, were mostly mild to moderate, and usually improved or resolved over time. The incidence of GI events was higher early in the course of treatment (primarily in the first month) and decreased over time. Overall, clinical trial discontinuations due to flushing and GI events were low.

Tecfidera may decrease lymphocyte counts in some patients. In clinical studies, mean lymphocyte counts decreased during the first year of treatment and then remained stable. The incidence of infections and serious infections was similar in Tecfidera-treated patients and those on placebo. There were no opportunistic infections in Tecfidera-treated patients. In patients with low lymphocyte counts, there was no increased incidence in serious infections. Patients taking Tecfidera should have a complete blood count (CBC) before starting treatment to measure lymphocyte counts. A follow up CBC is recommended annually and at the discretion of the treating physician.

“We are pleased to see a new, needed treatment option available to people living with MS,” said Dr. Timothy Coetzee, chief research officer at the National MS Society. “With the collaborative focus on MS research around the world, it is an exceptionally encouraging time for those who have been diagnosed with relapsing forms of MS.”

Tecfidera marks the fourth therapy Biogen Idec offers worldwide to people living with MS.

Tecfidera delayed-release capsules are indicated for the treatment of patients with relapsing forms of MS. Tecfidera has been proven to reduce MS relapses, progression of disability and MS brain lesions. The efficacy and safety of Tecfidera has been studied in a large, global clinical program with more than 3,600 MS patients, which includes an ongoing long-term extension study. It is believed that Tecfidera provides a new approach to treating MS by activating the Nrf2 pathway, although its exact mechanism of action is unknown. This pathway provides a way for cells in the body to defend themselves against inflammation and oxidative stress caused by conditions like MS.