Regulatory Focus™ > News Articles > New MS Drug Receives FDA Approval

New MS Drug Receives FDA Approval

Posted 13 September 2012 | By Alexander Gaffney, RAC 

The US Food and Drug Administration (FDA) has given approval to a new once-a-day drug intended to treat relapsing forms of multiple sclerosis (MS), the agency announced on 12 September.

Multiple sclerosis affects the connective tissue around the brain and spine, causing the immune system to attack the body's ability to communicate with itself. Though its causes remain unknown, patients are able to keep the disease in a state of remission through various treatment options.

Which is where Aubagio (teriflunomide), the newly approved drug manufactured by Massachusetts-based Genzyme, now a subsidiary of Sanofi, comes into play.

Aubagio represents an additional option for patients who have a relapsing version of the disease, explained Russell Katz, director of FDA's Division of Neurology Products. "In a clinical trial, the relapse rate for patients using Aubagio was about 30 percent lower than the rate for those taking a placebo," he said. "It's important to have a variety of treatment options available to patients."

The 30% reduction in the rate of patient relapse would make the drug less effective than two of its competitors, BG-12 and Gilenya, explained BioCentury. "In the Phase III TOWER trial, once-daily 14 mg Aubagio reduced the annualized relapse rate by 36.3% compared with placebo. Twice-daily 240 mg BG-12 reduced the endpoint by up to 53% in Phase III trials, while 0.5 mg once-daily Gilenya reduced it by up to 54%."

Drug Paired with Risk Mitigation Plan

The drug is not without risks to MS patients, though, cautioned FDA. Common side effects include diarrhea, abnormal liver tests and nausea. As a result, Aubagio will be paired with a Boxed Warning indicating the drug has been associated with fetal harm and potentially fatal liver problems. A Medication Guide will also be required to be dispensed with the drug, FDA said.

Some experts said that, despite the risks, some patients will still stand to benefit from the product. "Many people living with MS struggle with the additional burden of injectable therapies administered daily to weekly," said Dr. Aaron E. Miller, medical director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Mount Sinai Medical Center. Unlike previous MS treatments, Aubagio is taken orally in the form of a tablet.

"We are greatly encouraged to see a new oral therapeutic option become available to people living with MS," added Dr. Timothy Coetzee, chief research officer at the National MS Society.

The drug, though expensive at $45,000 a year, is still priced below its three nearest competitors, a Sanofi spokesman told Fierce Biotech.

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