Novartis presents data from FREEDOMS and FREEDOMS II trials showing effect of Gilenya (fingolimod) in NEDA4 measures of multiple sclerosis.

Novartis announced a new analysis from the phase III FREEDOMS and FREEDOMS II trials presented at the 67th American Academy of Neurology (AAN) Annual Meeting in Washington, DC, USA. These data showed that previously-treated patients with highly-active relapsing multiple sclerosis (RMS) who were treated with Gilenya (fingolimod) had a six-times greater likelihood of achieving 'no evidence of disease activity' across four key measures of disease activity compared to placebo over two years (odds ratio 6.35; 95% CI 3.02-13.35; p<0.0001). This is referred to as NEDA4 and is achieved when a patient with RMS has no relapses, no new MRI lesions, no MS-related brain shrinkage and no disability progression. This analysis was the first time patients with highly-active RMS who had been treated in the previous year with an injectable therapy were assessed using the NEDA4 definition that includes brain shrinkage. Brain shrinkage is a marker of the widespread inflammatory (diffuse) damage in the central nervous system and is associated with accumulated loss of function. By using this updated NEDA4 definition, physicians are able to get a more complete picture of a patient's disease and response to treatment, which is crucial to identify the optimal therapy to slow short- and long-term disease progression. This is especially important for people with highly-active RMS, who are at a greater risk of relapses and future loss of function, and may therefore require a different treatment approach.

Separate analysis from the entire phase III TRANSFORMS study also confirmed that after one year of treatment, RMS patients on Gilenya were twice as likely to achieve NEDA4 compared to patients given Avonex - interferon beta-1a i.m. injections (odds ratio 1.93; 95% CI 1.36-2.73; p=0.0002).

Comment: Gilenya, a sphingosine 1-phosphate receptor (S1PR) modulator, was the first oral treatment for multiple sclerosis, a condition that has been usually treated by injection or infusion. The drug has been effective in reducing the number of relapses and is the only approved treatment shown to consistently decrease brain volume loss. However concerns have been expressed about cardiovascular side effects of Gilenya which have required monitoring and Novartis has added to the label a caution that potential Gilenya patients should first take an ECG. An FDA alert highlighted a patient that developed the often fatal brain infection- progressive multifocal leukoencephalopathy. Gilenya was accepted by NICE on the basis of data confirming its benefit for patients with" highly active disease".