New Therapy for Relapsing MS Recommended for FDA Approval

An FDA advisory committee has recommended approval of FTY720 (fingolimod) for the treatment of relapsing multiple sclerosis (MS), a drug that, if approved, could be the first oral therapy for relapsing MS.

The advisory committee reviewed data from the largest clinical trial program ever submitted to the FDA as part of an MS new drug application and found that the data “provided evidence of superior efficacy of FTY720 over one of the most commonly prescribed treatments, interferon beta-1a IM (Avonex®), and to placebo, in reducing relapses and brain lesions (a measure of disease activity).” FTY720 also delayed disability progression, according to the researchers.

The safety profile of FTY720 has also been reviewed, with data from more than 4,500 patient years of experience, some of who are in their seventh year of treatment. In Phase III studies, “FTY720-related adverse events included transient, dose-related, generally asymptomatic heart rate reduction and infrequent transient AV conduction block at treatment initiation, mild (1-3 mm Hg) blood pressure increase, macular edema (more common with 1.25 mg than the 0.5 mg target dose), and generally asymptomatic, reversible elevation of liver enzymes.” Overall infection rates, including serious infections, were comparable among treatment groups, “although a slight increase in lung infections (primarily bronchitis) was seen in patients treated with FTY720.” Malignancies reported across the two studies were comparable.

FTY720 was granted priority review status in February of this year, reducing the standard 10-month review to six months. In May of this year, the FDA extended the priority review period for three months, until September 2010.

"This is an encouraging and important milestone for the MS community," said Dr. Patricia O'Looney, vice president of Biomedical Research at the National Multiple Sclerosis Society. "We believe that a treatment that reduces relapses and slows disability progression in a convenient oral formulation could encourage more people with MS to initiate treatment in the course of this life-long disease."