Late Friday afternoon, Novartis announced that the U.S. Food and Drug Administration (FDA) approved fingolimod (Gilenya) to treat relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older.
The news marks the first time that the FDA has approved a drug for the treatment of MS in pediatric patients. The drug was previously approved in the U.S. in 2010 for the treatment of adult patients with relapsing forms of MS, and it is proven to reduce relapses and delay disability progression. It was granted Breakthrough Therapy designation in the pediatric population in December 2017.
The approval is based on promising data collected from the Phase 3 PARADIGMS study, which enrolled 214 pediatric patients aged 10 to 17 and evaluated the safety and efficacy of fingolimod vs. interferon beta-1a in children and adolescents ages 10 or older with relapsing MS.
In the study, 86% of patients receiving Gilenya remained relapse-free after 24 months of treatment, compared to 46% of those receiving interferon beta-1a.
The Phase 3 was the first fulfilled randomized, controlled clinical trial specifically designed for pediatric relapsing MS. It revealed the drug’s ability to decrease the rate of annual relapse in this patient population for up to 2 years when compared to the ntramuscular injection.
“For the first time, we have an FDA-approved treatment specifically for children and adolescents with multiple sclerosis,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research in a press release
. “Multiple sclerosis can have a profound impact on a child’s life. This approval represents an important and needed advance in the care of pediatric patients with multiple sclerosis.”
Additionally, the study confirmed fingolimod’s favorable safety profile, demonstrating consistency with what has been seen in historical clinical trials that studied the drug in adults.
Serious risks for individuals who are administered the drug include slowing of heart rate, especially after the first dose. Fingolimod can also increase the risk of serious infections, and patients should continue to be monitored for infection during treatment and for 2 months after discontinuation of treatment. Progressive multifocal leukoencephalopathy (PML), a rare brain infection that usually leads to death or severe disability, has previously been reported in patients being treated with Gilenya.
Gilenya can also increase the risk for swelling and narrowing of the blood vessels in the brain, and other serious risks include: respiratory problems, liver injury, increased blood pressure and skin cancer.
Gilenya harm a developing fetus; women of child-bearing age should be advised of the potential risk to the fetus and to use effective contraception.
Gilenya must be administered with a patient Medication Guide that describes, in detail, important information about the drug’s uses and risks. The already-approved version of the drug is a once-a-day oral therapy that has been used to treat more than 215,00 patients in both clinical trials and in the post-marketing setting.
For more breaking news from the rare disease community, subscribe to Rare Disease Report