FDA Okays Fingolimod (Gilenya), First Treatment for Pediatric MS

Susan Jeffrey

May 11, 2018

The US Food and Drug Administration (FDA) has expanded approval for fingolimod (Gilenya, Novartis) to the treatment of children and adolescents with relapsing multiple sclerosis (MS) who are aged 10 years or older, the first such approval of a drug to treat MS in pediatric patients.

"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 a statement from the agency.

"Multiple sclerosis can have a profound impact on a child's life," Dunn adds. "This approval represents an important and needed advance in the care of pediatric patients with multiple sclerosis."

The FDA granted Priority Review and Breakthrough Therapy designation for this indication.

Fingolimod was first approved by the FDA in 2010 to treat adults with relapsing MS, and was the first oral medication approved for treatment of MS.

The current approval is based on results of the phase 3 PARADIGMS trial, presented last fall at the 7th Joint European Committee for Treatment and Research in Multiple Sclerosis-Americas Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS-ACTRIMS) 2017 meeting and reported at that time by Medscape Medical News.

PARADIGMS was a double-blind, randomized, multicenter study including 214 children and adolescents aged 10 to 17 years with a confirmed diagnosis of relapsing MS. They were randomly assigned to once-daily oral fingolimod (0.5 mg or 0.25 mg, dependent on body weight) or intramuscular interferon beta-1a once weekly.

Treatment with fingolimod resulted in large reductions in relapse rate, new MRI lesions, brain atrophy, and disability progression compared with interferon beta-1a.

"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 FDA announcement notes.

Side effects in pediatric trial participants were similar to those seen in adults, the statement says, with the most common side effects being headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain, and pain in extremities, the statement said.

Fingolimod must be dispensed with a patient Medication Guide, the FDA notes, "that describes important information about the drug's uses and risks."

Other points made in the release:

  • Serious risks of fingolimod include slowing of the heart rate, especially after the first dose.

  • Fingolimod may increase risk for serious infections; patients should be monitored for infection during treatment and for 2 months after discontinuation.

  • Progressive multifocal leukoencephalopathy (PML) has been reported in patients treated with fingolimod; cases usually occur in patients with weakened immune systems.

  • Fingolimod can cause vision problems.

  • Treatment may increase the risk for posterior reversible encephalopathy syndrome, swelling, and narrowing of the blood vessels of the brain.

  • Other serious risks include respiratory problems, liver injury, increased blood pressure, and skin cancer.

  • Fingolimod can cause harm to a developing fetus; women of child-bearing age should be advised of the potential risk to the fetus and to use effective contraception, the FDA statement concludes. 

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