Progressive Multiple Sclerosis Updates on Siponimod Trial and Ocrevus Approval
FDA Expands Approval of Gilenya to Treat Children With MS
MS disease-modifying drug is a first for use in pediatric patients.
By Becky Upham
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May 15, 2019
The Food and Drug Administration (FDA) announced approval of the drug Gilenya (fingolimod) on May 11 for the treatment of relapsing-remitting multiple sclerosis (RRMS) in children ages 10 and older, making it the first disease-modifying therapy approved for pediatric use. Approximately 98 percent of children with pediatric MS have the .
“As the first therapy specifically approved to treat children and teens with MS, this is a major milestone,” says Kathy Costello, associate vice president of healthcare access at the National Multiple Sclerosis Society and a nurse practitioner at Johns Hopkins Hospital in Baltimore.
Gilenya Effective in Reducing Flares
The first oral MS medication approved for adults, in 2010, Gilenya is a sphingosine 1-phosphate receptor modulator and is thought to work by retaining certain white blood cells in the lymph nodes, preventing them from crossing the blood-brain barrier into the central nervous system. Blocking the entry of these cells reduces inflammatory damage to the nervous system.
In December 2019, Gilenya was granted special “Breakthrough Therapy” designation by the FDA for the treatment of RRMS in children ages 10 years and older. This special category speeds the approval process for drugs and is reserved for therapies that offer “substantial improvement” over what’s currently available.
This designation, along with Gilenya’s final approval for use in children, was based on results of the PARADIGMS study, a double-blind, randomized, multi-center Phase III safety and efficacy trial of Gilenya vs. interferon beta-1a (an older MS treatment), specifically designed for people ages 10 to 17.
The PARADIGMS study has been presented at both the October 2019 ECTRIMS (European Committee for Treatment and Research in Multiple Sclerosis) meeting and the February 2019 ACTRIMS (Americas Committee for Treatment and Research in Multiple Sclerosis) forum in San Diego, California.
The primary objective of the study was to evaluate how successful the drugs were in preventing relapses, also called flares.
Among those taking Gilenya in the study, 86 percent remained relapse-free after 24 months of treatment, compared to 46 percent of those taking interferon beta-1a intramuscular injections. The safety profile of Gilenya in this study was similar to that seen in previous clinical trials in adults.
The reduction in relapses is especially notable, because the disease course in pediatric MS is suspected to be more inflammatory, resulting in more frequent relapses, than its adult counterpart.
“Repeated relapses are more common in young people with MS than in adults, so this is heartening news for patients and their families,” says Brenda Banwell, MD, chief of the division of neurology at Children's Hospital of Philadelphia, and coprincipal investigator of PARADIGMS in a release provided by Novartis, the maker of Gilenya.
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Off-Label Use in Children Is Fairly Common
Many doctors may have already been prescribing Gilenya “off-label” to children even before the approval. Off-label use is when a healthcare professional prescribes an FDA-approved drug for an unapproved disease, condition, or population.
According to FDA spokeswoman Sandy Walsh, this practice is fairly common, because most drugs prescribed for children have not been tested in children.
“By necessity, doctors have routinely given drugs to children ’off-label,’ which means the drug has not been approved for use in children based on the demonstration of safety and efficacy in adequate, well-controlled clinical trials,” Walsh says.
Even if it doesn’t dramatically change current prescribing habits, though, this approval is meaningful, says Walsh.
“This pediatric approval is important because now the drug labeling includes information describing safety, effectiveness, and dosing relating to use of the product in children,” she says.
MS Symptoms and Prevalence
MS occurs in the central nervous system (CNS) and causes symptoms throughout the body. It’s believed to be an autoimmune disorder, which means that instead of the body’s immune system attacking infections or germs as it should, it attacks and destroys healthy tissue.
In the case of MS, the immune system attacks the myelin sheath that protects nerve fibers in the CNS, causing inflammation and eventually destroying the protective layer, leaving scars that disrupt the electrical impulses (messages) between the brain and other parts of the body.
Estimates suggest that of the approximately 400,000 people in the United States with MS, 8,000 to 10,000 are children younger than 18. Children with MS generally have the same symptoms as adults with the disease, and can include difficulties balancing and walking, controlling the bladder or bowels, fatigue, weakness, and even depression.
Relapsing MS is characterized by symptom flares alternating with periods of remission. No two relapses are alike; a relapse can include new symptoms or symptoms that have been experienced before. The episode must last at least 24 hours to be considered a relapse, but it can last several weeks or even months.
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