Novartis announced 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.

"NEDA4 is a major step forward in assessing RMS progression, helping physicians to develop effective disease management and treatment strategies for their patients," said Vasant Narasimhan, global head of development at Novartis Pharmaceuticals.
"These data confirm that Gilenya's high efficacy across the four key measures is maintained in previously-treated highly-active RMS, and underscores the important role of Gilenya in the treatment of RMS patients."

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). The data provide further evidence of how Gilenya helps RMS patients achieve NEDA4 across four key measures of disease activity.

Multiple sclerosis (MS) is a chronic disorder of the central nervous system (CNS) that disrupts the normal functioning of the brain, optic nerves and spinal cord through inflammation and tissue loss.

The evolution of MS results in an increasing loss of both physical and cognitive (e.g. memory) function. This has a substantial negative impact on the approximately 2.3 million people worldwide affected by MS, a disease that most often begins in early adulthood.

People with MS can be diagnosed with relapsing forms of MS (RMS), which include relapsing remitting MS (RRMS) and secondary progressive MS (SPMS), or with primary progressive MS (PPMS).

The loss of physical and cognitive function in RMS is driven by two types of damage that result in the loss of neurons and brain tissue - distinct inflammatory lesions (referred to as focal damage), and more widespread inflammatory neurodegenerative processes (referred to as diffuse damage). Focal damage results in the loss of brain tissue and can clinically present as relapses.

Diffuse damage starts early in the disease, often goes unnoticed and is also associated with loss of brain tissue and accumulated loss of function.

Gilenya is the only oral disease-modifying therapy (DMT) to impact the course of relapsing MS (RMS) with high efficacy across four key measures of disease activity: relapses, MRI lesions, brain shrinkage (brain volume loss) and disability progression. Gilenya is approved in the US for first-line treatment of relapsing forms of MS in adults. In the EU, Gilenya is indicated for adult patients with highly-active relapsing remitting MS (RRMS) defined as either high disease activity despite treatment with at least one DMT, or rapidly evolving severe RRMS.

Gilenya targets both focal and diffuse CNS damage. It prevents cells that cause focal inflammation from reaching the brain (referred to as 'peripheral' action), but also enters the CNS and reduces the diffuse damage by preventing the activation of harmful cells residing in the CNS (referred to as 'central action').

It is important to address both focal and diffuse damage in RMS to effectively impact disease activity and help preserve an individual's functions.

The safety profile of Gilenya in RMS is well understood and based on substantial evidence from three major clinical trials and extensive real-world experience in more than 119,000 patients, with the total patient exposure now at approximately 218,500 patient years.

In addition to its ongoing development program for Gilenya in pediatric MS and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), the Novartis MS portfolio includes Extavia (interferon beta-1b for subcutaneous injection). Investigational compounds include BAF312, currently in phase III clinical development and being investigated as an oral therapy for secondary progressive MS (SPMS). Novartis is also exploring the IL-17 pathway in MS.