Biogen Idec wins FDA approval for MS pill that analysts project will become blockbuster drug

03/27/2013 4:19 PM

By Robert Weisman, Globe Staff


Federal regulators Wednesday approved Biogen Idec Inc.’s first pill to treat multiple sclerosis, a decision that cements the Weston biotechnology company’s dominance in the market for MS treatments and could allow thousands of patients to stop taking drugs by needle or through intravenous infusions.

The Food and Drug Administration said Biogen Idec can sell the treatment, called Tecfidera, in the United States in the form of a capsule to be taken twice a day. Analysts have projected Tecfridera could become one of the best-selling drugs of all time by the end of the decade if MS patients shift from existing therapies now on the market.

The company said it expects to start making the drug available within days.

MS, one of the most common causes of neurological disability in young adults, is an autoimmune disorder that affects the central nervous system of about 400,000 people in the United States and 2.5 million globally. It disrupts communication between the brain and other parts of the body. Most people experience their first MS symptoms between the age of 20 and 40.

“No drug provides a cure for multiple sclerosis so it is important to have a variety of treatment options available for patients,” Dr. Russell Katz, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Multiple sclerosis can impair movement, sensation, and thinking and have a profound impact on a person’s quality of life.”

Biogen Idec already markets two injectable MS drugs, Avonex and Tysabri.


http://www.boston.com/businessupdates/2013/03...odQ8SSg5LVOy1M/story.html

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Biogen's MS Drug Tecfidera Wins FDA Nod
By John Gever, Senior Editor, MedPage TodayPublished: March 27, 2013


SILVER SPRING, Md. -- Dimethyl fumarate has won FDA approval to treat relapsing-remitting forms of multiple sclerosis and will be sold under the name Tecfidera, the agency said Wednesday.
Manufactured by Biogen Idec, the drug is also known as BG-12. It becomes the third oral disease-modifying medication available to treat MS. Others include fingolimod (Gilenya) and teriflunomide (Aubagio).

Dimethyl fumarate has a novel mechanism of action relative to existing MS drugs. It is believed to activate the nuclear factor-like 2 transcriptional pathway, thereby reducing oxidative stress that contributes strongly to demyelination.

The drug has also been used for many years in Europe and elsewhere to treat psoriasis, and has also been used as a fungicide and desiccant in furniture manufacture.

Two placebo-controlled phase III trials served as the basis for approval, the agency said. Called DEFINE and CONFIRM, they enrolled a total of some 2,700 patients. In addition to comparing the drug with placebo, CONFIRM included a third arm with open-label glatiramer acetate (Copaxone), a standard injectable MS drug.

Both trials demonstrated that dimethyl fumarate was superior to placebo in preventing relapses and delaying disability progression. It also appeared to be somewhat more effective than glatiramer acetate in CONFIRM.

The most common adverse effects were gastrointestinal complaints and flushing. The FDA also noted that the drug carries a risk of lymphocytopenia, although infections did not appear to be increased as a result in the phase III studies.

"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 announcing the approval.

A Biogen competitor, Teva Pharmaceuticals, had petitioned the FDA to hold an advisory committee meeting on the drug before approving it, citing the hematologic effects and other risks, but the agency declined to do so.

http://www.medpagetoday.com/Neurology/MultipleSclerosis/38122