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FDA Expands Use of Gilenya® (fingolimod) for Children and Teens 10 Years and Older with Relapsing MS - First Approved Therapy for Pediatric MS

May 11, 2018

Updated 5/17/18
SUMMARY
  • 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.
  • For more information, contact the Gilenya Go Program online or by phone 1-800-GILENYA (1-800-445-3692) 
“As the first therapy specifically approved to treat children and teens with MS, this is a major milestone,” said Kathy Costello, MS, ANP-BC, MSCN, Associate Vice President of Healthcare Access at the National MS Society.
 
“The Society has been working on this issue for more than a decade, creating awareness and consistency around the diagnosis and care of children with MS,” said Cyndi Zagieboylo, President and CEO of the National MS Society. “We are a global leader in this field and are pleased to see the Society’s early and fundamental work helped lay the groundwork for this important breakthrough.”
 
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.
 
A Medication Guide describes potential side effects of Gilenya. Download the approved prescription information and Medication Guide.
 
For more information: Individuals may contact Novartis: 
1-800-GILENYA (8am-9pm Eastern). 
For people who qualify, patient assistance may be available from the Gilenya Go Program 1-800-GILENYA (1-800-445-3692).

FAQ About FDA Approval of Oral Gilenya for Children and Adolescents 10 and Older with Relapsing MS

Q. What is fingolimod/Gilenya?
A. Gilenya is a once daily oral treatment for relapsing MS. It works by binding to a docking site (sphingosine-1-phosphate receptor, or S1P receptor) on immune cells, including T cells and B cells that have been implicated in causing nervous system damage in MS. By binding to the docking site, Gilenya appears to force some immune cells to remain in lymph nodes and inhibits them from migrating into the brain and spinal cord.
 
Q. Should my child switch from his or her current therapy to Gilenya?
A. The decision about whether to take Gilenya should be made in collaboration with your child’s MS doctor, considering a variety of factors including the effectiveness of any therapy he or she is currently using, the potential risks and benefits, as well as costs and lifestyle factors.
 
Q. What was the Society’s role in the development of fingolimod as a treatment for pediatric MS?
A. 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. The Society’s early, fundamental work helped lay the groundwork for this important breakthrough.
  • 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.
  • 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. Several of the US Network of Pediatric MS Centers recruited participants for the trial.
 
Q. What types of MS is Gilenya be approved to treat?
A. The FDA has expanded the approved use of Gilenya to include the treatment of adults and now children and adolescents 10 years of age and older who have relapsing forms of MS. In other words, people who experience periodic MS attacks, such as those who have relapsing-remitting MS or secondary-progressive MS with relapses.
 
Q. How is Gilenya taken?
A. Gilenya comes in capsules. It is taken by mouth once a day with or without food. The dose in children may be determined according to body weight.
 
Q. How effective is Gilenya in children and adolescents?
A. 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. These results were reported at the ECTRIMS MS meeting in 2017. In MRI results reported at several medical meetings, fingolimod significantly reduced disease activity on MRI scans, including the rate of brain volume loss.
 
Q. What are the potential side effects of Gilenya?
A. 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), a swelling and narrowing of the blood vessels in the brain (posterior reversible encephalopathy syndrome) and progressive multifocal leukoencephalopathy (PML), a rare brain infection that can lead to death or severe disability. Other serious risks include lung problems, liver injury, increased blood pressure and skin cancer. Patients should be monitored for infection during treatment and for two months after discontinuation of treatment.
 
A medication guide will help inform patients and their families about its use and potential risks. The Medication Guide further describes potential side effects and symptoms that those taking Gilenya should pay attention to. 
 
Q. Has Gilenya been proven to be “better” than other disease-modifying therapies for children and adolescents with MS?
A. So far, the only thorough comparison to an existing disease-modifying therapy has been in the phase 3 clinical trial comparing Gilenya to Avonex® (interferon beta-1a, Biogen Idec). This trial suggested short-term superiority of Gilenya, but the trial was too short to be definitive in terms of comparing long-term benefits of the two therapies.
 
Q. How long would a person take Gilenya?
A. There is no specified time limit for taking Gilenya.
 
Q. Will children or adolescents taking Gilenya have to get any special medical tests or monitoring?
A. Yes. Monitoring will likely be similar to that recommended for adults. Because there is the potential for significant side effects in some patients, the prescribing information recommends pre-treatments tests including:
  • a new or recent blood test to establish lymphocyte (immune cell) count;
  • an eye (ophthalmologic) evaluation;
  • a new or recent blood test to evaluate liver enzyme levels;
  • a new or recent electrocardiogram in those using heart medications, those who have cardiac risk factors, or those who on examination have slow or irregular heart beat prior to starting Gilenya;
  • those who do not have a history of chickenpox or vaccination against varicella zoster virus (VZV) should be tested for VZV antibodies, and those who are negative should consider vaccination before starting treatment with Gilenya.
  • Individuals should be observed for six hours after the first dose is taken in the doctor’s office for signs and symptoms that may be associated with decreased heart rate and other potential heart effects. In addition, the doctor is likely to recommend an eye exam 3 to 4 months after starting Gilenya and may order occasional blood tests and evaluate vision during routine visits. Patients should be monitored for infection during treatment and for two months after discontinuation of treatment.
Q. What does Gilenya cost?
A. The price of Gilenya depends on the provisions of insurance coverage and the degree to which that individual will be eligible for programs designed to assist with out-of-pocket costs.
 
Q. Will my health insurance cover Gilenya?
A. Coverage will depend on individual insurance plans. For people who qualify, assistance may be available from the Gilenya Go Program 1-800-GILENYA (1-800-445-3692)
 
Q. Where can I get information about the support that Novartis will provide?
A. Physicians and people with MS can contact:
Gilenya Go Program 1-800-GILENYA (1-800-445-3692)
 
Q. Are other disease-modifying therapies being tested in children and adolescents with MS?
A. Yes, clinical trials of disease-modifying therapies are ongoing, including the following: teriflunomide; dimethyl fumarate (this study is recruiting participants); and alemtuzumab (this study is recruiting).
 
 
Avonex is a registered trademark of Biogen Idec
Gilenya is a registered trademark of Novartis AG
 
 
 

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. 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. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease. MS affects more than 2.3 million people worldwide.

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