A drug known as Gilenya (fingolimod), which was first approved in 2010 for the treatment of Multiple Sclerosis (MS) in adult patients, was recently re-approved by the U.S. Food and Drug Administration as the first official treatment option for relapsing form of multiple sclerosis (MS) in children and adolescents age 10 years and older.
“For the first time, we have an FDA-approved treatment specifically for children and adolescents with multiple sclerosis,” explains Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “Multiple sclerosis can have a profound impact on a child’s life. This approval represents an important and needed advance in the care of pediatric patients with multiple sclerosis.”
MS is an autoimmune disease of the central nervous system (CNS) caused by a disruption between the brain and other parts of the body. MS is characterized by chronic and inflammatory phenotypes. It is the most common neurological disability in young adults, occurring more frequently in women than men. For many individuals with MS, periods of worsening function and appearance of new symptoms, known as relapses or flare-ups, begin by recovery periods or remissions. Over time, recovery may not be completely fulfilled, this leads to the progressive stage of MS consisting of a decrease in function and an increase in disability. Furthermore, many with MS will experience their first symptoms, such as vision problems or muscle weakness, between the ages of 20 to 40 and about two to five percent will have symptoms onset before age 18. The estimates suggest that 8,000 to 10,000 pediatric-aged individuals in the U.S. have MS.
The effectiveness of Gilenya in treating pediatric patients was evaluated by comparison to another MS drug (interferon beta-1a) in a clinical trial involving 214 patients between the ages of 10 to 17. The results showed that 86% of patients receiving Gilenya were MS relapse-free after two years of treatment in comparison to 46% of those patients administered the interferon beta-1a.
In pediatric patients, Gilenya possessed side effects which were quite similar to those exhibited in adults. The most common symptoms included headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities.
The administration of Gilenya consists of dispensation with a patient Medication Guide that details crucial information about the use and risk of the drug. The serious risks include a slow heart especially after the first dose of Gilenya. Additionally, Gilenya may increase the risk of infection particularly a form of brain infection known as progressive multifocal leukoencephalopathy (PML). It can also cause harm to the developing fetus, lead to vison complications, and serious respiratory problems.