FDA approved fingolimod (Gilenya, Novartis) for the treatment of children and adolescents aged 10 years to less than 18 years who have relapsing forms of multiple sclerosis (MS)—the first disease-modifying therapy indicated for these patients.
This approval expands the age range for Gilenya, which was previously approved for patients aged 18 years and older with relapsing MS.
“We now finally have an FDA-approved treatment for children and adolescents with relapsing MS,” said Dr. Brenda Banwell, chief of the Division of Neurology at Children's Hospital of Philadelphia, co-principal investigator of the study that supported the pediatric approval. “Repeated relapses are more common in young people with MS than in adults, so this is heartening news for patients and their families.”
While the drug will not be commercially available for purchase, it will be available through the Gilenya Go Program—provided to patients who have less than 40 kg in body weight, Novartis said in a statement provided to FormularyWatch.
Gilenya’s U.S. list price before discounts and rebates to insurers is $7,857 a month. “Most commercially insured patients taking Gilenya pay a $0 copay per month, and our medical copay support program can help pay for certain medical tests before starting Gilenya. Novartis is also exploring ways to extend a variety of existing programs for adults to pediatric patients to help those who do not have insurance or have difficulties affording their medication,” Novartis said.
In addition, Novartis is in discussions with US health insurance providers regarding coverage for the pediatric indication.
While MS is mostly diagnosed in adults, children and adolescents with the chronic disease often experience more frequent relapses and brain lesions than adults with MS.
“We have eagerly anticipated a proven treatment option for younger people living with MS,” said Elin Phillips, president of the Pediatric Multiple Sclerosis Alliance, in a Novartis statement. “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.”
The approval of Gilenya for the younger patient population was supported by a double-blind, randomized, multi-center phase 3 safety and efficacy study of fingolimod versus interferon beta-1a, designed specifically for children and adolescents with RMS. The study demonstrated that fingolimod reduced the rate of relapses (annualized relapse rate) by approximately 82% over a period of up to 2 years, compared to interferon beta-1a intramuscular injections in children and adolescents (ages 10 and older) with relapsing MS.