May 16, 2018 PR-M05-18-NI-060
EAST HANOVER, N.J., May 11, 2018 /PRNewswire/ -- Novartis today announced that the US Food and Drug Administration (FDA) has approved Gilenya® (fingolimod) for the treatment of children and adolescents 10 to less than 18 years of age with relapsing forms of multiple sclerosis, making it the first disease-modifying therapy indicated for these patients2.
This approval expands the age range for Gilenya, which was previously approved for patients aged 18 years and older with RMS. Gilenya was granted Breakthrough Therapy designation by the FDA in December of 2017 for this pediatric indication.
"We now finally have an FDA-approved treatment for children and adolescents with relapsing MS," said Dr. Brenda Banwell, Chief of the Division of Neurology at Children's Hospital of Philadelphia, who served as co-Principal investigator of the pivotal study that supported the pediatric approval. "Repeated relapses are more common in young people with MS than in adults, so this is heartening news for patients and their families."
While MS is mostly diagnosed in adults, children and adolescents with the chronic disease often experience more frequent relapses and brain lesions than adults with MS1.
"We have eagerly anticipated a proven treatment option for younger people living with MS," said Elin Phillips, President, Pediatric Multiple Sclerosis Alliance. "Until now, there have been no large clinical trials to assess treatment efficacy of disease-modifying therapies in children and adolescents. On behalf of the pediatric community, we would also like to thank the families and the clinical trial patients who made this progress possible."
The approval of Gilenya for the younger patient population was supported by PARADIGMS, a double-blind, randomized, multi-center Phase III safety and efficacy study of fingolimod vs. interferon beta-1a, designed specifically for children and adolescents with RMS4. The primary endpoint demonstrated that fingolimod reduced the rate of relapses (annualized relapse rate) by approximately 82% (p <0.001) over a period of up to two years compared to interferon beta-1a intramuscular injections in children and adolescents (ages 10 and older) with relapsing MS2. The safety profile of Gilenya in this study was overall consistent with that seen in previous clinical trials in adults2. In this study, while more adverse events (AEs) were reported in the interferon beta-1a group, severe AEs were reported at a higher frequency in fingolimod-treated patients2.
"After becoming the first approved oral MS therapy in the US in 2010, we're proud this new approval represents another first for Gilenya," said Fabrice Chouraqui, US President of Novartis Pharmaceuticals Corporation. "It is the latest achievement in our ongoing commitment to drive innovation in MS and bring additional treatment options to more patients, including young people with MS."
About the Phase III PARADIGMS Study
The Phase III PARADIGMS study (NCT01892722) is a flexible duration (up to two years), double-blind, randomized, multi-center study to evaluate the safety and efficacy of oral fingolimod compared to interferon beta-1a in children and adolescents with a confirmed diagnosis of MS, followed by a five-year open label extension phase5. The study enrolled 215 children and adolescents with MS, 10 to less than 18 years of age with an Expanded Disability Status Scale (EDSS) score between 0 and 5.55. Patients were randomized to receive once-daily oral fingolimod (n=107, 0.5 mg or 0.25 mg, dependent on patients' body weight) or intramuscular interferon beta-1a (n=108) once weekly5.
The primary endpoint of the study was the frequency of relapses in patients treated up to 24 months (annualized relapse rate)5. Secondary endpoints include the number of new or newly enlarged T2 lesions, Gadolinium enhancing T1 lesions, safety and the pharmacokinetic properties of fingolimod, all measured throughout the treatment period5.
The Phase III PARADIGMS study was conducted in 87 sites over 26 countries, and was designed in partnership with the US Food and Drug Administration, European Medicines Agency and the International Pediatric Multiple Sclerosis Study Group.
About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic disorder of the central nervous system (CNS) that disrupts the normal functioning of the brain, optic nerves and spinal cord through inflammation and tissue loss6. In adults, there are three types of MS: relapsing-remitting MS (RRMS), secondary progressive MS (SPMS) and primary progressive MS (PPMS)7. Approximately 85 percent of people with MS have relapsing-remitting MS, where the immune system attacks healthy tissue8. In children, RRMS accounts for nearly all cases (approximately 98 percent)9.
In the US, MS affects around 400,000 people10.
About Gilenya (fingolimod) in Adults
Gilenya was the first once-a-day pill approved to treat relapsing multiple sclerosis (MS). Approved for first-line use, Gilenya is a disease-modifying therapy (DMT) that offers freedom from injections, which may fit many patients' lifestyles. Gilenya decreases the frequency of MS flare-ups (relapses) and helps slow down the physical problems caused by RRMS.
Gilenya is the most prescribed oral once-daily DMT in the US, with approximately 79,800 patients having been exposed to Gilenya. Worldwide, Gilenya has been used to treat approximately 231,000 patients in both clinical trials and the post-marketing setting, with approximately 536,000 years of patient experience3.
GILENYA is a prescription medicine used to treat relapsing forms of multiple sclerosis (MS) in adults and children 10 years of age and older.
Important Safety Information
You should not take GILENYA if in the last 6 months you experienced heart attack, unstable angina, stroke or mini-stroke (transient ischemic attack or TIA), or certain types of heart failure. Do not take GILENYA if you have an irregular or abnormal heartbeat (arrhythmia), including a heart finding called prolonged QT as seen on an ECG, or if you take medicines that change your heart rhythm. Do not take GILENYA if you are allergic to fingolimod or any of the other ingredients.
GILENYA may cause serious side effects such as:
GILENYA may harm your unborn baby. Talk to your doctor if you are pregnant or planning to become pregnant. Women who can become pregnant should use effective birth control while on GILENYA, and for at least 2 months after stopping. If you become pregnant while taking GILENYA, or within 2 months after stopping, tell your doctor right away. It is not known if GILENYA passes into breast milk. Talk to your doctor about the best way to feed your baby if you take GILENYA. A pregnancy registry is available for women who become pregnant during GILENYA treatment. For more information, contact the GILENYA Pregnancy Registry by calling Quintiles at 1-877-598-7237, by e-mailing email@example.com, or by going to www.gilenyapregnancyregistry.com.
Tell your doctor about all your medical conditions, including if you had or now have an irregular or abnormal heartbeat; stroke or mini-stroke; heart problems; a history of repeated fainting; a fever or infection, or if you are unable to fight infections due to a disease or are taking medicines that lower your immune system, including corticosteroids, or have taken them in the past; eye problems; diabetes; breathing or liver problems; or uncontrolled high blood pressure. Also tell your doctor if you have had chicken pox or have received the chicken pox vaccine. Your doctor may test for the chicken pox virus, and you may need to get the full course of the chicken pox vaccine and wait 1 month before starting GILENYA. Children 10 years and older should complete their vaccination schedule before starting GILENYA.
If you take too much GILENYA, call your doctor or go to the nearest hospital emergency room right away.
Tell your doctor about all the medicines you take or have recently taken, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
The most common side effects with GILENYA were headache, abnormal liver tests, diarrhea, cough, flu, sinusitis, back pain, abdominal pain, and pain in arms or legs.
In the pediatric study:
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Please see full Prescribing Information, including Medication Guide, at: https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/gilenya.pdf
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SOURCE Novartis Pharmaceuticals Corporation
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Located in East Hanover, NJ Novartis Pharmaceuticals Corporation is an affiliate of Novartis which provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2017, the Group achieved net sales of USD 49.1 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 124,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world.