Novartis International AG announced today that oral FTY720 (fingolimod) was able to significantly reduce relapse rates and slow disability progression over two years in a large-scale, phase 3 trial involving 1,272 people with relapsing-remitting MS. According to a company press release, safety data confirmed a positive benefit-risk profile for the lower of two doses tested, and the company plans to submit applications to drug regulatory agencies for marketing approval of the potential therapy at the end of 2009.
Background: FTY720 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 an earlier phase 2 study led to several large-scale phase 3 trials. Initial positive results from the TRANSFORMS study, comparing two different doses of fingolimod with Avonex® (interferon beta-1a, Biogen Idec) over only one year were presented at the American Academy of Neurology meeting in spring 2009. Adverse side effects seen more often in the fingolimod treatment groups in this trial included temporary reductions in heart rate at the start of therapy, small increases in blood pressure, and a few cases of macular edema (swelling of the back of the eye). Two deaths from herpes infections occurred in the group taking the higher dose of fingolimod, and seven cases of localized skin cancer occurred in the fingolimod groups.