The Food and Drug Administration has approved a new oral drug for the treatment of multiple sclerosis (MS).
Manufactured by the Cambridge, Massachusetts-based Biogen Idec (NASDAQ: BIIB), twice-a-day capsules of dimethyl fumarate will be sold under the brand name Tecfidera and should be available within the coming weeks. Biogen has not disclosed the price of the drug, but like other MS medications, monthly treatments are expected to cost tens of thousands of dollars.
The drug becomes the third oral medication available to treat MS, joining fingolimod, which is sold by Novartis (NYSE: NVS) under the brand name Gilenya, and teriflunomide, which is marketed under the brand name Aubagio by the French drug maker Sanofi (NYSE: SNY).
Before the introduction of oral medications in 2011, the only drugs available to treat MS were injectable.
“With the FDA approval of Tecfidera, we will offer the MS community a treatment with strong efficacy and a favorable 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 Scangos, CEO of Biogen.
The FDA said it based its approval on two placebo-controlled phase III trials that involved a total of some 2,700 patients. In those studies, dimethyl fumarate was superior to a placebo in preventing relapses and delaying disability progression. In tests that involved glatiramer acetate (Copaxone), an injectable MS drug, Tecfidera appeared to be somewhat more effective.
“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.
According to Biogen, Tecfidera significantly reduces disease activity, including relapses and development of brain lesions, and demonstrated a favorable safety and tolerability profile.
FDA approval of Tecfidera comes just one week after a ruling that gave the pharmaceutical company a 15-year patent on the drug, helping protect its market exclusivity and effectively holding off the entry of generic competition longer than previously anticipated.
The drug has been used for many years in Europe and elsewhere to treat psoriasis. When doctors in Germany noted relapses ended in two MS patients taking the dimethyl fumarate for psoriasis, researchers took another look at the drug.
“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.”
The most common side effects of dimethyl fumarate are skin flushing, diarrhea, nausea and abdominal pain, usually within the first month. The FDA noted the drug also carries a risk of lymphocytopenia, although infections did not appear to be increased as a result in the phase III studies.
Because of those hematologic effects and other risks, Teva Pharmaceuticals (NYSE: TEVA), the manufacturer of Copaxone, petitioned the FDA to hold an advisory committee meeting on the drug before approving it, but the agency declined to do so.
Multiple sclerosis is a chronic, often disabling disease that attacks the central nervous system, destroying the protective myelin sheath that protects nerve cells in the brain, optic nerve and spinal cord.
Symptoms may be mild or severe, ranging from numbness in the limbs to paralysis or loss of vision. The progression, severity and symptoms of MS are unpredictable and vary from one person to another.
An estimated 400,000 Americans have the disease and more than 2 million worldwide. There is no known cure.