FDA approves first drug to treat MS in children

The FDA on Friday approved Gilenya for relapsing multiple sclerosis in children and adolescents aged 10 years and older — the first approval of a drug to treat MS in pediatric patients, the agency announced in a press release.

“For the first time, we have an FDA-approved treatment specifically for children and adolescents with multiple sclerosis,” Billy Dunn, MD, director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research, said in the 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.”

Gilenya (fingolimod, Novartis), an immunosuppressive drug, was first approved by the FDA in 2010 to treat adults with relapsing MS. Approximately 2% to 5% of people with MS have symptom onset before age 18 years, and estimates suggest that 8,000 to 10,000 children and adolescents in the United States have MS, according to the FDA.

The FDA granted priority review and breakthrough therapy designation for this indication in December, based on data from the phase 3 PARADIGMS study, which evaluated the safety and efficacy of fingolimod vs. interferon beta-1a in children and adolescents with relapsing MS. The randomized controlled trial demonstrated that treatment with fingolimod resulted in an 82% reduction in the rate of relapses (annualized relapse rate) in children and adolescents for up to 2 years compared with interferon beta-1a intramuscular injection (P < .001), according to results released by Novartis in December. The safety profile of fingolimod was consistent with clinical trials in adults. The most common side effects were headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities, according to the FDA.

Fingolimod must be dispensed with a patient medication guide that describes important information about the drug’s uses and risks. Serious risks include slowing of the heart rate, especially after the first dose, and fingolimod may increase the risk for serious infections. The The agency said patients should be monitored for infection during treatment and for 2 months after discontinuation of treatment.

In 2015, the FDA issued a warning of possible infection for those prescribed fingolimod, following reports of progressive multifocal leukoencephalopathy in patients prescribed the drug, usually in those with weakened immune systems. Fingolimod can cause vision problems and may increase the risk for posterior reversible encephalopathy syndrome. Other serious risks include respiratory problems, liver injury, increased blood pressure and skin cancer.

Disclosure: Dunn reports no relevant financial disclosures.