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Fingolimod Granted “Breakthrough Therapy Designation” for Pediatric MS by the FDA

December 19, 2017

Novartis has announced that oral fingolimod (Gilenya®, Novartis) has been granted “Breakthrough Therapy designation” by the U.S. Food and Drug Administration (FDA) for the treatment of children and adolescents 10 years of age or older with relapsing  MS. This designation will expedite the review process now that Novartis has filed for approval of fingolimod to treat pediatric MS.
Breakthrough Therapy designation can speed up the development and review of therapies intended to treat a serious condition when clinical evidence indicates that the treatment may demonstrate substantial improvement over available therapy. No treatment is currently approved by the FDA to treat children and adolescents with MS.
The designation was granted to fingolimod based on phase III trial results presented at the European Committee for Treatment and Research in MS in October 2017. In the largest clinical trial yet in children and adolescents with MS, fingolimod reduced the annual number of relapses by 82% over two years, compared to treatment with interferon beta-1a (Avonex®). After two years, 86% of those on fingolimod had not experienced a relapse, compared to 39% of the Avonex group. The trial also showed benefits measured by MRI. Side effects were similar to what have been seen in the adult population.
Fingolimod was approved by the FDA in 2010 for adults with relapsing forms of MS. Fingolimod is called a sphingosine 1-phosphate receptor modulator, which is thought to act by retaining certain white blood cells in the lymph nodes, preventing them from crossing the blood-brain barrier into the central nervous system. Preventing the entry of these cells reduces inflammatory damage to the nervous system.
Read more about pediatric MS
Read more information about the FDA’s approaches to making such drugs available as rapidly as possible
Avonex is a registered trademark of Biogen
Gilenya is a registered trademark of Novartis AG 

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system. Symptoms range from numbness and tingling to blindness and paralysis, and there is currently no cure for MS. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. An estimated 1 million people live with MS in the United States. Most people with MS are diagnosed between the ages of 20 and 50, and it affects women three times more than men.


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