Novartis Announces FDA Approval of Gilenya as the First Disease-Modifying Therapy for Pediatric Relapsing Multiple Sclerosis

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 (RMS), making it the first disease-modifying therapy indicated for these patients.

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 MS.

“Since revolutionizing the treatment of relapsing MS as the first oral disease-modifying therapy, Gilenya has become an important mainstay of treatment for adult patients,” said Paul Hudson, CEO, Novartis Pharmaceuticals. “Today’s announcement is a result of our pioneering approach and ongoing commitment to advancing care for all individuals living with MS, and we are delighted this has led to a long-awaited, specifically-approved treatment option for young patients.”

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 Gilenya vs. interferon beta-1a, designed specifically for children and adolescents with RMS[4]. The primary endpoint demonstrated that Gilenya 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 MS. The safety profile of Gilenya in this study was overall consistent with that seen in previous clinical trials in adults.

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 Gilenya compared to interferon beta-1a in children and adolescents with a confirmed diagnosis of multiple sclerosis (MS), followed by a five-year open label extension phase. 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 5.5. Patients were randomized to receive once-daily oral Gilenya (0.5 mg or 0.25 mg, dependent on patients’ body weight) or intramuscular interferon beta-1a once weekly.

The primary endpoint of the study was the frequency of relapses in patients treated up to 24 months (annualized relapse rate). Secondary endpoints include the number of new or newly enlarged T2 lesions, Gadolinium enhancing T1 lesions, safety and the pharmacokinetic properties of Gilenya, all measured throughout the treatment period.

The 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.

SOURCE: Novartis