Novartis will present new data that reinforce generally early and sustained efficacy benefit and long-term safety profile for Gilenya (fingolimod), the first once-daily oral therapy approved to treat relapsing forms of multiple sclerosis (MS). The data will be presented at the 28th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

"As the first once-daily oral MS therapy, growing real-world experience reinforces Gilenya's high efficacy and long-term safety profile," said David Epstein, Head of the Pharmaceuticals Division of Novartis Pharma AG. "With data showing an early treatment effect on relapses and brain volume loss, Gilenya continues to show positive outcomes for patients and Novartis remains committed to addressing the significant remaining unmet medical need in the MS community."

A new post hoc analysis of two large phase III studies shows treatment with Gilenya 0.5 mg led to significant benefits on relapse-related outcomes within the first three months and on brain volume loss by six months compared to placebo.

There was a significant (p<0.05) Gilenya treatment effect on time to first confirmed relapse within three months in both the pivotal FREEDOMS study (n=1272), and FREEDOMS II, the second large phase III placebo-controlled study (n=1083). The differences between Gilenya and placebo became persistently significant by Day 82 in FREEDOMS and Day 64 in FREEDOMS II, respectively.

Furthermore, in the FREEDOMS study, patients-treated with Gilenya 0.5 mg had on average a 35 per cent reduction in brain volume loss compared with placebo at the first MRI evaluation after six months of treatment (mean percent brain volume change of -0.22 for Gilenya vs. -0.34 for placebo; p=0.006). In FREEDOMS II, there was a 39 per cent reduction in brain volume loss (mean percent volume change of - 0.23 for Gilenya vs. - 0.38 for placebo; p=0.012) at six months.

"Understanding the onset of efficacy is an important consideration in the treatment of MS as early effective treatment may improve patient outcomes," said Professor Ludwig Kappos, MD, Chair of Neurology, University Hospital, Basel, Switzerland. "The new analysis of phase III data shows a significant early effect of Gilenya on relapses and MRI measures, and further supports its role as a valuable treatment option for relapsing-remitting MS."

First results from the PANGAEA observational study in Germany indicate that 90 per cent of investigators and 91 per cent of patients rated Gilenya treatment success, defined as efficacy and tolerability, as "Good" or "Very Good". PANGAEA is a German registry study aimed to enroll a total of 4,000 patients with a follow-up of five years designed to investigate the efficacy and safety of Gilenya in everyday clinical practice. As of May 2012, one year after initiation of the registry, more than 1,850 patients were enrolled in 475 participating centers. These results also showed an overall safety profile in line with previously reported data.

In addition, a separate analysis of time to discontinuation of therapy among MS patients receiving Gilenya and other disease modifying treatments (DMTs) using pharmacy claims in the US (n=1891) show Gilenya-treated patients were significantly less likely to discontinue treatment over the 12 month observation period (Gilenya: 27.8 per cent, other DMT cohorts: 42.7-54.5 per cent; p<0.01) and discontinued later than patients using injectable DMTs.

Results from more than 3,500 patients further characterize long-term safety profile.

A new integrated analysis of safety data from phase II, phase III and study extensions for fingolimod (all doses, n=3553) show a safety profile generally consistent with previous results. The total fingolimod exposure was 9,070 patient years, with 1,510 patients treated for more than three years and some for more than seven years. As of August 2012, more than 49,000 patients have been treated with fingolimod in clinical trials and in the post-marketing setting, and there is approximately 52,000 patient years of exposure.

Gilenya, licensed from Mitsubishi Tanabe Pharma Corporation, is the first in a new class of compounds called sphingosine 1-phosphate receptor (S1PR) modulators. It has demonstrated superior efficacy compared to Avonex, a commonly prescribed treatment, showing a 52 per cent relative reduction in annualized relapse rate (primary endpoint) and a 40 per cent relative reduction in the rate of brain atrophy (secondary endpoint) at one year in a pivotal head-to-head trial in patients with relapsing-remitting multiple sclerosis. In a recent post hoc sub-group analysis, Gilenya showed a 61 per cent relative reduction in annualized relapse rate compared to interferon-beta-1a (IM) at one year in subgroups of patients with highly active relapsing-remitting MS not responding to interferon treatment.

Gilenya is a generally highly effective once-daily oral MS treatment. In clinical trials it was generally well tolerated with a manageable safety profile, and there is increasing experience of Gilenya's long-term effectiveness and safety profile.

In clinical trials, the most common side effects were headache, liver enzyme elevations, influenza, diarrhea, back pain, and cough. Other Gilenya-related side effects included transient, generally asymptomatic, heart rate reduction and atrioventricular block upon treatment initiation, mild blood pressure increase, macular edema, and mild bronchoconstriction. The rates of infections overall, including serious infections, were comparable among treatment groups, although a slight increase in lower respiratory tract infections (primarily bronchitis) was seen in patients treated with Gilenya. The number of malignancies reported across the clinical trial program was small, with comparable rates between the Gilenya and control groups.