Novartis pediatric multiple sclerosis drug Gilenya shows significant effect in phase III trial

Results showed an 82% reduction in the rate of relapses (annualized relapse rate) over a period of up to two years with oral Gilenya compared to treatment with interferon beta-1a intramuscular injections (p <0.001)¹. Gilenya is not currently approved in Canada, or elsewhere, for the treatment of pediatric MS.

"We are pleased to contribute to the advancement of research in MS by conducting and announcing the results of this first controlled, randomized clinical trial specifically in pediatric patients. These findings add to the knowledge available in this population where there is a high unmet need," said Dawn Bell, Chief Scientific Officer, Novartis Pharmaceuticals Canada Inc.

PARADIGMS is the first controlled, randomized trial specifically designed for pediatric MS. The results were presented at the 7th Joint European and Americas Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS-ACTRIMS) meeting in Paris.

Canada has the highest rate of MS in the world, with the disease affecting more than 100,000 Canadians. While MS is most often diagnosed between the ages of 15 and 40, younger children and older adults are also diagnosed³.

"The results from PARADIGMS provide important new information," said Dr. Daniela Pohl, pediatric neurologist, Associate Professor of Pediatric Neurology at the University of Ottawa, and one of the study investigators. "Pediatric MS is challenging to manage because until now, we have been extrapolating data from clinical trials with adult patients to children."

Additional data from the study demonstrated¹:

A significant reduction in the number of new / newly enlarging T2 and Gd-T1 lesions in the brain in Gilenya-treated patients compared to those treated with interferon beta-1a, as measured by magnetic resonance imaging (MRI).

Individuals treated with Gilenya had significantly less brain shrinkage (measured by MRI as brain volume loss), compared to those treated with interferon beta-1a.

The safety profile of Gilenya was overall consistent with that seen in previous clinical trials.

Gilenya (fingolimod) is indicated as monotherapy for the treatment of adult patients with the relapsing-remitting form of multiple sclerosis (MS) to reduce the frequency of clinical exacerbations and to delay the progression of physical disability. Gilenya is generally recommended in MS patients who have had an inadequate response to, or are unable to tolerate, one or more therapies for multiple sclerosis⁴.

The Phase III PARADIGMS study (NCT01892722) is a flexible duration (up to two years), double-blind, randomized, multi-center study to evaluate the safety and efficacy of oral Gilenya compared to interferon beta-1a in children and adolescents with a confirmed diagnosis of multiple sclerosis (MS), followed by a five-year open label extension phase.

 The study enrolled 215 children and adolescents with MS, between the ages of 10 and 17 years with an Expanded Disability Status Scale (EDSS) score between 0 and 5.5. Patients were randomized to receive once-daily oral Gilenya (0.5 mg or 0.25 mg, dependent on patients' body weight) or intramuscular interferon beta-1a once weekly⁵.

The primary endpoint of the study was the frequency of relapses in patients treated up to 24 months (annualized relapse rate). Secondary endpoints include the number of new or newly enlarged T2 lesions, Gadolinium-enhancing T1 lesions, safety and the pharmacokinetic properties of Gilenya, all measured throughout the treatment period⁵.

The Phase III PARADIGMS study was conducted at 87 sites in 25 countries, including Canada, and was designed in partnership with the US Food and Drug Administration, European Medicines Agency and the International Pediatric Multiple Sclerosis Study Group.