Oct. 5, 2011 -- People with multiple sclerosis (MS) may soon have a second needle-free option to control their disease.
Last year, the FDA approved the first disease-modifying pill, a drug called Gilenya, to treat MS.
Now a new study shows that a different drug, a once-daily pill called teriflunomide, may also slow the progression of the neurological disease and its disabling attacks better than a placebo.
Currently, most of the disease-modifying drugs that treat MS are given by injection or intravenous infusion.
"Some patients have been sitting on the sidelines waiting for effective and safe oral medications," says researcher Jerry S. Wolinsky, MD, a professor of neurology at the University of Texas Health Science Center in Houston. Others with MS have been giving themselves regular injections for more than a decade.
"Their skin is just not holding up well. It's harder and harder for me to convince them to keep doing this because of the difficulties they have with these long-term injections," Wolinsky tells WebMD. For those reasons, he says, pills that work as effectively as the shots are "very important" options.
And drugmakers are racing to bring them to market.
In addition to teriflunomide, three other oral medications have been granted fast-track reviews by the FDA.
Testing a New Pill to Control MS
The new study, which is published in the New England Journal of Medicine, enrolled nearly 1,100 patients in 21 countries.
Ninety percent had the relapsing remitting form of MS -- an early stage of the disease. In this stage there are occasional flare-ups typically followed by partial or complete recovery of function.
The patients enrolled had at least two relapses in the previous two years, but no relapses in the two months before the study. Nearly 800 patients completed the two-year study.
The study found that teriflunomide reduced relapses in MS patients by 31% compared to a placebo. At the highest dose, the drug significantly reduced the number of treated patients who experienced worsening disability. It also reduced areas of active inflammation in the brain compared to placebo.
"People on the drug had fewer attacks," says researcher Paul O'Connor, MD, professor of neurology at the University of Toronto. "So what it meant to a patient is that if you were destined to have three attacks in one year, you would actually only have two."
"It slowed relapse and reduced the risk of disability progression by about 30%," O'Connor says.
The drug works by preventing fast-growing immune system cells from activating, multiplying, and responding to the body's own proteins, causing inflammation.
Rates of serious infections, a common risk of drugs that subdue an overactive immune system, were similar between the placebo and treatment groups. Serious infections affected 2.2% of those taking the placebo, 1.6% of those on the lower dose of teriflunomide, and 2.5% on the higher dose.
There were three cases of serious kidney infections in people taking the higher drug dose. One led a person to quit the study.
Because the drug interferes with fast-growing cells, researchers say women who are pregnant or planning a pregnancy should not take the medication.
Anita Burrell, a vice president at Sanofi-Aventis, the drug's manufacturer, says the company has applied to the FDA for review. They should know this month if the agency has accepted their application.
"We know cost is a big issue in the MS environment. But it's clearly too premature at this point to speculate for this product in particular," she says.
MS drugs are some of the most expensive therapies on the market. A study published earlier this year in Neurology found that the health gains MS patients get from their medications come at extremely high prices.
Gilenya, the pill that's already available to patients, costs $4,000 a month, or $48,000 annually. By comparison, the injectable drug Copaxone costs between $2,800 and $3,200 a month.
Experts who were not involved in the study say they think there's a good chance it will be approved by the FDA.
"It's my opinion that this drug needs to be on the market," says Jack Burks, MD, a neurologist in Reno, Nev., and chief medical officer of the Multiple Sclerosis Association of America. "People should be given the opportunity to take this drug. Whether they take it or not should be up to the patient and the doctor."
He says teriflunomide appears to be effective and safe, at least in the short term.
"We don't know the long-term safety. Therefore we're cautiously optimistic that the long-term safety data will be good and people who don't want to take shots anymore won't have to," Burks says.
"Just because it's a pill doesn't mean it's safer," he says.