Fingolimod, an investigational daily multiple sclerosis (MS) pill, has achieved priority review by the Food and Drug Administration (FDA). Following submission of a new drug application (NDA) in December 2009, the US regulatory body has, for the first time, granted priority review of an oral MS drug. Fingolimod has the potential to answer an unmet clinical need.
Priority review, granted to medicines with high unmet need or in areas with inadequate available treatments, means that the review time will be six months instead of ten. It is hoped fingolimod will be available in the US this year. Fingolimod was submitted for European review at the end of 2009.
“We welcome the decision granting priority review to fingolimod, which underscores the potential benefits of this medicine to patients,” said Trevor Mundel, MD., Global Head of Development at Novartis Pharma AG. “MS is a leading cause of neurological disability in young adults, particularly in women, and this medicine has the potential to offer real advances in the care of people with MS.”
The submission was based on positive data for fingolimod, which was recently published in the New England Journal of Medicine (NEJM). Data from the Phase III pivotal studies, FREEDOMS and TRANSFORMS, confirm the significant efficacy of fingolimod in reducing relapses and disability progression in patients with relapsing-remitting MS – the most common form of the disease.
Fingolimod has a well-studied safety profile, with over 5,300 patient years of exposure. The fingolimod MS study programme includes more than 4,000 patient years of exposure, with some patients in the sixth year of therapy, making it the largest Phase III clinical trial programme ever conducted in MS. This robust clinical trial programme strengthens the potential for fingolimod to be the first approved product in a new therapeutic class called S1P receptor modulators.
As stated in the NEJM, an available oral treatment option for patients with MS is highly desirable to improve convenience, diminish side effects and improve compliance. Fingolimod’s novel mode of action also means that its suppressive effect on the immune system can be reversed if treatment is stopped, allowing circulating lymphocytes to regain normal levels within weeks
