Novartis Receives FDA Breakthrough Therapy Designation for Pediatric MS

Mathew Shanley

On Monday, Novartis announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for fingolimod for the treatment of children and adolescents 10 years of age or older with relapsing multiple sclerosis (MS).

Fingolimod is currently being marketed at Gilenya, and was approved in the U.S. in 2010 for the treatment of adult patients with relapsing forms of MS. It is proven to reduce relapses and delay disability progression.

The National MS Society estimates that 8,000-10,000 children up to 18 years old in the U.S. have been diagnosed with MS, and that another 10,000-15,000 have experienced at least one symptom suggestive of the condition. Diagnosing the disease in a patient’s youth is more difficult than it is in adults because the symptoms are like that in other childhood disorders.

This week’s designation is the result of promising data collected from the Phase 3 PARADIGMS study, which evaluated the safety and efficacy of fingolimod vs. interferon beta-1a in children and adolescents ages 10 or older with relapsing MS.

"Despite the fact that children experience approximately two to three times as many relapses as a typical adult onset MS patient, there are currently no disease-modifying therapies approved for the pediatric population," said principal investigator of the PARADIGMS study Dr. Tanuja Chitnis, Director of the Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital, Boston, U.S., and Scientist, Ann Romney Center, Brigham and Women's Hospital, Boston, U.S. in a press release. "Children with MS differ from adults in important ways and additional treatment options for pediatric patients are needed.”

The Phase 3 was the first completed randomized, controlled clinical trial specifically designed for pediatric relapsing MS. It discovered the drug’s ability to reduce the rate of annual relapse in this patient population for up to 2 years when compared to interferon beta-1a intramuscular injection.

"We're proud of this regulatory milestone, which represents part of our commitment to advance treatment options for young people with MS," said Fabrice Chouraqui, President of Novartis Pharmaceuticals Corporation. "Novartis is looking forward to working with the FDA to bring a therapy with a long track record in adults with relapsing MS to this younger patient population as soon as possible."

The study also confirmed fingolimod’s favorable safety profile, exhibiting consistency with what has been seen in previous clinical trials studying the drug in adults.

The already-approved version of the drug is a once-a-day oral therapy that has been used to treat more than 215,00 patients in both clinical trials and in the post-marketing setting.
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