New Multiple Sclerosis Drug Nearing FDA Approval

New Multiple Sclerosis Drug Nearing FDA Approval

A promising new oral drug to treat Multiple Sclerosis (MS) is one step closer to being approved by the Food and Drug Administration. Results from two Phase III clinical trials published in the New England Journal of Medicine  show that that dimethyl fumarate (BG-12) capsules appear to significantly reduce relapses and disease activity by as much as 40 percent.

The drug’s manufacturer, Biogen Idec (NYSE: BIIC) says that the results of the separate, two-year clinical studies demonstrate significant and meaningful reductions in MS relapses and brain lesions in patients with relapsing-remitting multiple sclerosis, as well as benefits in slowing the progression of the disease.

“The data from its clinical development program consistently indicate that dimethyl fumarate (BG-12) may provide tangible benefits and address existing treatment needs of people living with MS. “ said Dr. Katherine Dawson, senior medical director of Biogen Idec’s Neurology Research and Development.

If approved, the drug would be only the third oral medication available to treat the chronic disease.   Just last week, the FDA green lighted the sale of teriflunomide, which is marketed under the brand name Aubagio by the French drug maker Sanofi (NYSE: SNY). The first MS pill, fingolimod, which is sold by Novartis (NYSE: NVS) under the brand name Gilenya, hit the market two years ago.

“Because MS is a chronic disease, we look for treatment options that not only control relapses but also slow patients’ disease progression for as long as possible,” said Ralf Gold, PhD, chair of the Department of Neurology at St. Josef-Hospital/Ruhr-University in Bochum, Germany and lead author of one of the studies.  “Dimethyl fumarate demonstrated efficacy, as well as positive safety and tolerability profiles, which is a very attractive combination for an MS treatment.”

In both studies, more than 1,000 patients from 28 countries, ages 18 to 55, were given either BG-12 or a placebo.  In one of the studies, the injectable MS drug glatiramer acetatemedication (Copaxone) was added to the mix.

What the researchers discovered was that on average, patients getting BG-12 went from 72 weeks to more than 90 weeks before experiencing a relapse, compared with 30 and 38 weeks among those assigned a placebo. And though the study wasn’t designed to compare the drug to Copaxone, which is sold by Teva Pharmaceutical (NYSE: TEVA), BG-12 did appear to be slightly more effective than the injectable medication.

The path that led to dimethyl fumarate being used to combat MS began 15 years ago in Germany, where the drug was approved for the treatment of psoriasis. When doctors noted that relapses in two MS patients taking the medication for psoriasis appeared to cease, researchers took another look at the drug.

BG-12 is not without side effects, which include skin flushing, nausea, vomiting and diarrhea, usually in the first month.

“Relapsing MS is a disease that we have treatments for, but they’re really on two ends of the spectrum,” Dr. Robert Fox, a neurologist at the Cleveland Clinic’s  Mellen Center for MS and a lead author in one of the studies, told CNN.  “We have injection treatments that have modest efficacy. They reduce relapses by about 30 percent.

“And we have very effective therapies, but they are infusion therapies and they have risk – risks of a brain infection – that may be fatal. This new treatment offers the opportunity for sort of the best of both worlds, in that it has very good efficacy, but it’s in the form of a pill and appears to have much reduced risk than other therapies.”

In both studies, researchers looked at the effectiveness of taking BG-12 either two or three times a day. Between the two studies, taking the pill three times a day wasn’t significantly better than twice a day. The approval application submitted to the FDA calls for twice daily dosing.

Both Fox, and Timothy Coetzee, chief medical research officer for the National MS Society, say the studies’ results look very promising and that they believe the FDA will approve the drug. The FDA has until December 28 to make a decision.

If approval is given, Biogen should announce the brand name it will use to sell BG-12 (dimethyl fumarate), as well as the price. For many MS patients, cost is a factor. Currently, the price of Aubagio is about $45,000 per year and for Gilenya it’s about $48,000.

MS is a chronic disease which attacks the body’s central nervous system and destroys the myelin sheath that protects the nerve cells. An estimated 400,000 Americans have the disease; more than 2 million worldwide. There is no known cure.

Authored by: Richard Lenti