FDA approves teriflunomide for patients with relapsing forms of MS

September 13, 2012

FDA has approved teriflunomide (Aubagio, Genzyme, a Sanofi company), a once-daily, oral treatment indicated for patients with relapsing forms of multiple sclerosis.

FDA has approved teriflunomide (Aubagio, Genzyme, a Sanofi company), a once-daily, oral treatment indicated for patients with relapsing forms of multiple sclerosis (MS).

Teriflunomide is an immunomodulator with anti-inflammatory properties. Although the exact mechanism of action for teriflunomide is not fully understood, it may involve a reduction in the number of activated lymphocytes in the central nervous system. It has shown significant efficacy across key measures of MS disease activity, including reducing relapses, slowing the progression of physical disability, and reducing the number of brain lesions as detected by MRI.

The FDA approval was based on efficacy data from the TEMSO (TEriflunomide Multiple Sclerosis Oral) trial. In the phase 3 TEMSO trial, teriflunomide 14 mg significantly reduced the annualized relapse rate (P=.0005) and the time to disability progression (P=.0279) at 2 years versus placebo in patients with relapsing forms of MS. Aubagio 7 mg significantly reduced the annualized relapse rate (P=.0002) in the trial.

Fingolimod (Gilenya, Novartis) was FDA approved in 2010, and at the time was the only oral, once-a-day disease modifying therapy for relapsing MS.

“Many people living with MS struggle with the additional burden of injectable therapies administered daily to weekly,” Aaron E. Miller, medical director, The Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Mount Sinai Medical Center, said in a Genzyme press release. “The FDA’s approval of Aubagio, a new oral treatment option, is an encouraging advancement for the MS community and may be a valuable treatment for people living with this often debilitating disease.”

The ongoing teriflunomide clinical development program, involving more than 5,000 patients in 36 countries, is among the largest of any MS therapy. Some patients in extension trials have been treated for up to 10 years.

The teriflunomide label includes a boxed warning citing the risk of hepatotoxicity and teratogenicity (based on animal data).

In MS clinical studies with teriflunomide, the incidence of serious adverse events were similar among teriflunomide and placebo-treated patients. The most common adverse events associated with teriflunomide in MS patients included increased ALT levels, alopecia, diarrhea, influenza, nausea, and paresthesia.

The labeling for teriflunomide was also informed by the estimated 2.1 million years of patient exposure globally since the launch of leflunomide, which is indicated in the United States for the treatment of rheumatoid arthritis. Teriflunomide is the principal active metabolite of leflunomide. Severe liver injury including fatal liver failure has been reported in patients treated with leflunomide.

As part of its commitment to MS patients, Genzyme has developed the MS One to One program. MS One to One provides information about multiple sclerosis, teriflunomide, and other relevant resources. Information and support are also available here.

According to the National Multiple Sclerosis Society, approximately 400,000 Americans have MS, and every week about 200 people are diagnosed. Worldwide, MS affects about 2.1 million people. Because the Centers for Disease Control and Prevention does not require U.S. physicians to report new cases, and because symptoms can be completely invisible, the numbers can only be estimated.