• The U.S. Food and Drug Administration has approved the expansion of the use of the oral MS therapy Gilenya® (fingolimod, Novartis AG) to include the treatment of children and adolescents 10 years of age or older with relapsing MS.
• This is the first therapy specifically approved to treat pediatric MS.
• A large phase 3 clinical trial showed benefits in reducing relapses and disease activity on MRI.
• The most common side effects were headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities. A Medication Guide will help inform patients and their families about its use and potential risks.

DETAILS

The U.S. Food and Drug Administration has approved Gilenya® (fingolimod, Novartis AG) for use in the treatment of children and adolescents 10 years of age or older with relapsing MS, making it the first therapy approved for the treatment of pediatric multiple sclerosis.

About Pediatric MS: Some estimates suggest that up to five percent of cases of MS begin prior to adulthood. Pediatric MS generally manifests as active relapsing-remitting disease. For more than a decade, the National MS Society has been a global leader in creating awareness and consistency around the diagnosis and care of children with MS. In 2004 the Society supported the development of the International Pediatric MS Study Group, which led to the publication of many papers establishing best practices for diagnosing and caring for children with MS. In 2006 the Society established a U.S. Network of Pediatric MS Centers, a research network of twelve medical centers focusing on understanding and better treating this disease, and providing infrastructure and centralized data collection to facilitate current and future research studies.

About Gilenya: Gilenya was approved by the FDA in 2010 for adults with relapsing forms of MS. Gilenya 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. Gilenya comes in capsules. It is taken by mouth once a day with or without food.

Potential Benefits for Children with Relapsing MS: This approval is based on phase 3 trial results, originally presented at the European Committee for Treatment and Research in MS in October 2017. Fingolimod reduced the annual number of relapses by 82% over two years, compared to treatment with interferon beta-1a (Avonex®, Biogen). After two years, 86% of those on fingolimod had not experienced a relapse, compared to 39% of the Avonex group. In MRI results reported at medical meetings, fingolimod significantly reduced disease activity on MRI scans, including the rate of brain volume loss.

Potential Risks: Side effects seen in the phase 3 clinical trial in children and adolescents were similar to what have been seen in the adult population. The most common side effects were headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities. The prescribing information carries warnings about other potentially serious risks, including heart events on first dose, infections, an eye problem called macular edema, increases of liver enzymes (which could indicate liver injury) and progressive multifocal leukoencephalopathy (PML), a rare brain infection that can lead to death or severe disability. A medication guide will help inform patients and their families about its use and potential risks.