FDA Gives Fingolimod a Breakthrough Therapy Designation for Relapsing MS in Children

The U.S. Food and Drug Administration has given fingolimod a Breakthrough Therapy designation as a treatment for children 10 years and older and adolescents with relapsing multiple sclerosis.

Novartis is marketing it in the United States under the brand name Gilenya for adults with relapsing MS. It has yet to approved for children, however.

The Breakthrough Therapy designation is expected to accelerate regulators’ review of fingolimod as a treatment for younger patients.

“We’re proud of this regulatory milestone, which represents part of our commitment to advance treatment options for young people with MS,” Fabrice Chouraqui, president of Novartis Pharmaceuticals, said in a press release. “Novartis is looking forward to working with the FDA to bring a therapy with a long track record in adults with relapsing MS to this younger patient population as soon as possible.”

FDA based the designation on findings from the Phase 3 PARADIGMS study (NCT01892722), which compared fingolimod’s safety and effectiveness with Avonex’s (interferon beta-1a) in children with relapsing MS.

The trial involved 215 patients, aged 10 to 17, at 87 medical centers in 25 countries. All of the participants had had the disease a short time — a mean of 1.2 years — and it was very active.

Researchers randomly assigned the children to one of two doses of fingolimod once a day or a  weekly injection of Avonex for up to two years. The fingolimod dose the children received was based on their body weights.

The children who took fingolimod had 82 percent fewer MS flare-ups than those who received Avonex, the study showed. In addition, fingolimod delayed patients’ brain deterioration and the progression of their functional disability.

Fingolimod’s safety profile was similar to that in clinical trials of adults, with no additional concerns. The most common adverse events were upper respiratory tract infections, fever, and increased susceptibility to flu and influenza-like illnesses.

“Despite the fact that children experience approximately two to three times as many relapses as a typical adult-onset MS patient, there are currently no disease-modifying therapies approved for the pediatric population,” said Dr. Tanuja Chitnis, director of the Partners Pediatric Multiple Sclerosis Center at Massachusetts General Hospital.

“Children with MS differ from adults in important ways and additional treatment options for pediatric patients are needed,” added Chitnis, the principal investigator of the PARADIGMS trial.

Gilenya helps control four measures of relapsing MS activity in adults: relapse rate, brain lesions, brain volume loss, and disability progression. Lesions are areas where the myelin sheath that protects nerve cells has deteriorated.