Dr. Patricia O'Looney - FDA Hearing - Fingolimod

National MS Society

A U.S. Food and Drug Administration advisory committee today recommended that the agency approve marketing of fingolimod capsules (formerly called Gilenia, Novartis International AG) for the treatment of relapsing multiple sclerosis. If approved, fingolimod would be the first oral disease-modifying therapy for the treatment of MS. While the FDA is not required to follow the recommendations of its advisory committees, it usually does. According to Novartis, the agency is expected to make a final decision about whether to approve the drug in September 2010.

Among its discussions, the advisory committee recommended that fingolimod be approved at the dose (0.5 mg once daily) recommended by Novartis and that:
• Fingolimod demonstrated substantial evidence of effectiveness for the treatment of relapsing MS to reduce the frequency of clinical relapses and to delay the accumulation of physical disability;
• the safety data currently known justify the drug’s approval, and the FDA should require a post-marketing study that would proactively gather information about adverse events and longer-term safety, the effects on a broader range of people than were included in the trials, and possible complications of taking other medications including steroids along with fingolimod;
• patients should be monitored during the first dose for possible lowering of heart rate and other potential heart effects, and that some assessments for potential adverse events related to eye (especially macular edema) and lung function be required, to an extent to be determined by the FDA;
• the FDA should consider requiring a study to evaluate whether a lower dose would be as effective as the recommended dose, with fewer adverse events;
• this therapy should be approved as a first-line therapy, meaning that patients would be eligible to take fingolimod without having to try an alternative therapy first.

About the drug: Fingolimod is a new class of therapy in development for treating multiple sclerosis. It binds to a docking site (sphingosine-1-phosphate receptor, or S1P receptor) on immune cells, including T cells and B cells, that have been implicated in causing nervous system damage in MS. The drug appears to induce immune cells to remain in lymph nodes, where they can do little harm, preventing them from migrating into the brain and spinal cord.

Positive results from two large-scale phase III clinical trials have been published showing that fingolimod significantly reduced multiple sclerosis relapse rates. One of the trials also suggested that fingolimod could slow the progression of disability. (New England Journal of Medicine January 20, 2010.) Read more about these studies on our Website. In December 2009, Novartis applied to the FDA and European regulators for marketing approval of this compound for the treatment of relapsing MS.