FDA approves new Biogen MS drug

The Biogen Idec manufacturing plant in Cambridge.Globe File/2008/Boston Globe

Federal regulators on Wednesday approved Biogen Idec Inc.’s first pill to treat multiple sclerosis, cementing the Weston company’s dominance in MS treatments and potentially allowing thousands of patients to stop taking their drugs by needle or intravenous infusions.

Biogen Idec already markets two of the best-selling injectable MS treatments and had long sought Food and Drug Administration approval to sell the new drug, called Tecfidera, in the United States.

Most crucially, the FDA approved Tecfidera as a “first-line” treatment, meaning doctors can prescribe it for newly diagnosed patients as well as those already taking MS drugs. Analysts say Tecfidera could become one of the top-selling prescription drugs of all time by the end of the decade if large numbers of MS patients shift from current treatments.

“This is huge,” said Acton resident Susan Cohn-Child, a financial­ manager in Boston who was diagnosed with MS about 17 years ago and has been treating it with drug injections.

“Would I prefer to take a pill? Yeah, absolutely,” she said. “The injections work for me, so I’m going to keep doing it for now. But it’s life-changing for people who are needle-phobic or who can’t tolerate injections.”

Biogen Idec expects to make the drug — a capsule to be taken twice daily — available to patients within days. It will also soon disclose the price of the treatment, expected to be tens of thousands of dollars a month per patient, the bulk of it covered by insurance in most cases.

“Tecfidera is a great advance for patients,” said Tony Kingsley, executive vice president at Biogen Idec. “From the Biogen standpoint, this fits into a portfolio of MS treatments for patients with different needs. It puts us in an incredible position in the marketplace.”

FDA officials disclosed their approval shortly before financial markets closed. Shares of Biogen Idec jumped nearly 3.2 percent to $182.68, an increase of $5.59.

MS, one of the most common causes of neurological disability in young adults, is a chronic autoimmune disorder that affects the central nervous systems 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 symptoms between the ages of 20 and 40 and over time suffer from muscle weakness, loss of balance, and a progressive decline in function.

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

In a pair of clinical trials, patients taking Tecfidera had fewer MS relapses than others taking a placebo, according to the FDA. One of the trials showed patients’ disabilities worsened less frequently when they took Tecfidera rather then a placebo.

Biogen Idec said the capsule 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.”

The FDA warned the drug may decrease a patient’s white blood cells, which help protect the body from infection, but it said no significant increase in infections was found in patients taking Tecfidera during the trials. The FDA recommends that patients’ white blood cell count be assessed before they start treatment.

People using Tecfidera reported some redness of the skin and stomach problems such as nausea, vomiting, and diarrhea, but those side effects may decrease over time, the FDA said. Its approval did not come with a so-called black box warning on the label — the agency’s most serious caution.

“It’s a best-case scenario in terms of the label they received from the FDA,” said Mark Schoenebaum, biopharmaceutical analyst for the investment research firm ISI Group in New York. “There’s no black box, and there’s no requirement for onerous monitoring.”

Schoenebaum said Biogen Idec can say Tecfidera is safer and more effective than two other oral MS therapies that beat it to the market: Gilenya, made by the Swiss drug giant Novartis AG, which has its global research headquarters in Cambridge; and, Aubagio, marketed by the Cambridge-based Genzyme division of the French pharmaceutical company Sanofi SA.

Analysts have said Tecfidera, which has yet to be approved in Europe, could generate annual global sales of $4 billion by 2018 and between $3 billion and $6 billion by the end of the decade.

The drug is being reviewed by regulators in Canada, Australia, and Switzerland.

Sales at the upper end of the projection range would put Tecfidera’s revenue close to that of some of the best-selling drugs ever, such as the heartburn pill Nexium and the cholesterol medicine Lipitor.

Biogen Idec last year rang up sales of $2.9 billion from its lead MS drug, Avonex, approved by the FDA in 1996, and $1.1 billion from its MS drug Tysabri, which started being sold in 2004. Tysabri, which prevents nerve damage in patients with relapsing MS, was pulled from the market in 2005 after some patients died from a rare brain infection but was relaunched in 2006. The company has since designed a test to predict which patients are vulnerable to the infection.

Kingsley said Biogen Idec employees were celebrating at the company’s Weston and Cambridge sites Wednesday afternoon when they learned of the FDA’s decision.

“There’s buzzing in the hallway,” he said. Then noting that the company continues to develop two other MS treatments, he added, “But we’re getting right back to work.”