Full results from the positive phase 3 PARADIGMS study, investigating the safety and efficacy of fingolimod (Gilenya, Novartis), an oral disease-modifying therapy, versus interferon beta-1a in children and adolescents (ages 10–17 years) with multiple sclerosis (MS), have been announced. Treatment with fingolimod resulted in an 82% reduction in the rate of relapses (annualized relapse rate) over a period of up to two years compared with interferon beta-1a intramuscular injections (P < 0.001). PARADIGMS is the first controlled, randomized trial specifically designed for pediatric MS.
"Pediatric MS patients experience more frequent relapses and are more likely to accumulate physical disability at an earlier age than patients diagnosed as adults," said Tanuja Chitnis, MD, Principle Investigator for PARADIGMS and Director of the Partners Pediatric Multiple Sclerosis Center at Massachusetts General Hospital and a scientist at the Ann Romney Center at Brigham and Women's Hospital, both in Boston. "Yet, current therapies are limited to drugs that have not been tested in a controlled manner in this age group. PARADIGMS was uniquely designed for this patient population. Its results signify an important step towards a potential new treatment that could improve the lives of these young patients."
The fingolimod-treated patients demonstrated a significant reduction in the number of new/newly enlarging T2 and Gd-T1 lesions in the brain compared with those treated with interferon beta-1a, as measured by magnetic resonance imaging (MRI). The number and volume of lesions are associated with increased relapses and disability progression.
In addition, patients treated with fingolimod had significantly less brain shrinkage (measured by MRI as brain-volume loss), compared with those treated with interferon beta-1a. Brain shrinkage in adults is associated with the loss of physical and cognitive function.
The safety profile of fingolimod was consistent overall with that seen in previous clinical trials, with more adverse events reported in the interferon group.
In an additional analysis, fingolimod significantly delayed disability progression, defined as confirmed disability progression (CDP), compared to interferon beta-1a.
Fingolimod is not currently approved for the treatment of pediatric MS. Novartis is working on submission with health authorities worldwide. It is currently approved in the U.S. for the first-line treatment of relapsing forms of MS in adults, and in the European Union 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.
The results in pediatric/adolescent patients were presented at the 7th Joint European and Americas Committee for Treatment and Research in Multiple Sclerosis meeting on October 28, 2017 in Paris, France.
Source: Novartis; October 28, 2017.