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 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. 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.