Gilenya’s Clinical Trial Results Presented at ECTRIMS Reinforce Long-Term Efficacy Profile in RMS Patients

On October 8, 2015, data on two Phase III clinical trials called FREEDOMS and FREEDOMS II on Gilenya (fingolimod), a drug developed by Novartis, were presented at the 31st Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), currently being held in Barcelona, Spain (October 7-10). Results from the data analysis reinforced the long-term efficacy profile of Gilenya in patients with relapsing multiple sclerosis (RMS).

Multiple sclerosis (MS) is a disease that damages the brain through inflammation and tissue loss, disturbing the brain’s normal functioning and preventing an effective communication between brain regions. An average of 2.3 million people live with MS worldwide, and the disease is considered one of the most common neurological diseases and major cause of disability in young adults. It is believed that MS is caused by an abnormal immune response and environmental factors such as infections, cigarette smoking and exposure to hazard chemicals. Signs and symptoms of MS may include difficulties in movement/coordination, muscle weakness/spasms, problems with speech/swallowing, and impairment in vision, among others. RMS patients experience attacks and worsening of the symptoms, along with a physical and cognitive dysfunction that results from loss of neurons/brain tissue, distinct inflammatory lesions, and widespread inflammatory neurodegenerative processes.

There is no cure for MS, but a large number of medications are currently available on the market, having different mechanisms of action. Among them, an oral disease-modifying drug called Gilenya (fingolimod) is approved in the U.S. as a first-line treatment for RMS in adults. Its efficacy was evaluated with four key measures: relapses, MRI lesions, brain shrinkage (brain volume loss) and disability progression. Gilenya has been used to treat upwards of 125,000 patients and the overall benefit risk profile of the drug was ranked positive. However, the long-term efficacy of Gilenya in RMS patients is unknown.

At the ECTRIMS 2015 congress, pooled data from the FREEDOMS and FREEDOMS II core and extension trials on Gilenya were analyzed by looking at a new evaluation criteria called ‘no evidence of disease activity’ (NEDA-4). Basically, NEDA-4 is performed at a yearly basis and for the Gilenya clinical trials was extended to over seven years. NEDA-4 is defined as having: no relapses, MS-related brain shrinkage, MRI lesions, and disability progression over a certain period of time.

Results showed that during the first year, 27.1% of the patients treated with Gilenya accomplished NEDA-4 compared to 9.1% on placebo. Furthermore, when RMS patients switched from placebo to Gilenya after the second year, the proportion of patients who accomplished NEDA-4 doubled in year three from 12.7% to 27.4%. Finally, among patients on continuous Gilenya treatment, 31.2% to 44.8% achieved NEDA-4 in the years three to seven.

Based on the results, the team suggested that instead of evaluating the three standard parameters (relapses, MRI lesions and disability progression), it is recommended that physicians and specialists use the NEDA-4 method to anticipate brain shrinkage and long-term disability outcomes. Also, NEDA-4 evaluation is advised during the first year as it is a good predictor of disability and brain shrinkage over the subsequent five years.

In summary, the findings reinforced further the long-term efficacy profile of Gilenya in patients with RMS, and highlighted the importance of NEDA-4 in assessing RMS, facilitating the prediction of long-term consequences of disease progression in MS patients.

“MS is a chronic debilitating disease and these data are important in showing the long-term efficacy of Gilenya, and the importance of early treatment to help improve long-term outcomes for patients,” said Vas Narasimhan, Novartis Global Head of Development in a press release. “Better understanding of the course of a person’s MS through assessment of NEDA-4 can help physicians identify the optimal, effective treatment approach as early as possible for their patients.”