Gilenya Approved by FDA as First DMT for Children, Ages 10 and Older, with Relapsing MS

Alice Melão, MSc avatar

by Alice Melão, MSc |

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Gilenya (fingolimod) has become the first disease-modifying therapy approved by the U.S. Food and Drug Administration (FDA) to treat children and adolescents with relapsing forms of multiple sclerosis (MS).

This expanded approval allows Gilenya, previously indicated for adults patients 18 or older, to be used to treat pediatric relapsing MS patients starting at age 10.

The FDA decision was supported by data from the ongoing Phase 3 PARADIGMS study (NCT01892722) comparing the safety and effectiveness of Gilenya to Avonex (interferon beta-1a) in these young patients.

“We now finally have an FDA-approved treatment for children and adolescents with relapsing MS,” Brenda Banwell, MD, chief of the division of Neurology at Children’s Hospital of Philadelphia and co-principal investigator of PARADIGMS study, said in a press release. “Repeated relapses are more common in young people with MS than in adults, so this is heartening news for patients and their families.”

Marketed in the United States and Europe by Novartis, Gilenya has been shown to help control MS activity in adults, including relapse rate, brain lesions, brain volume loss, and disability progression. The FDA named it a breakthrough therapy in late 2017 to advance its development for children with relapsing MS.

PARADIGMS enrolled 215 pediatric MS patients, ages 10 to 18, at 87 sites across 26 countries. The children were randomly assigned for up to two years to one of two doses of oral Gilenya each day  — either 0.5 or 0.25 mg — or to a weekly intramuscular injection of Avonex. (Long-term Gilenya use is now under study in children continuing in trial’s open-label extension.)

Early data revealed the Gilenya treatment could reduce by almost 82% the frequency of MS flares in pediatric patients compared to Avonex.

A later, additional analysis also showed that Gilenya — in comparison to interferon beta-1a— lowered patients’ annualized relapse rate by 85.8%, the  development of new brain lesions by 53.4%, and reduce by 77.2% the risk of three-month confirmed disability progression.

“We have eagerly anticipated a proven treatment option for younger people living with MS,” said Elin Phillips, president of the 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.”

Gilenya safety profile in younger MS patients was similar to that reported in clinical trials of adult patients, with no additional concerns. The most common side effects were upper respiratory tract infections, fever, and an increased susceptibility to the flu and flu-like illnesses.

“After becoming the first approved oral MS therapy in the U.S. in 2010, we’re proud this new approval represents another first for Gilenya,” said Fabrice Chouraqui, Novartis’ U.S. president. “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.”