Silver Spring, Maryland, USA: The U.S. Food and Drug Administration (FDA) on Friday expanded the use of Swiss drugmaker Novartis AG’s relapsing multiple sclerosis (MS) drug Gilenya to treat children 10 years of age or older.
The approval of Gilenya (fingolimod) for use in the treatment of children and adolescents with relapsing MS, makes it the first therapy approved for the treatment of pediatric multiple sclerosis.
Multiple sclerosis is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body and is among the most common causes of neurological disability in young adults.
Gilenya is the first FDA-approved drug to treat pediatric patients suffering from relapsing MS, the U.S. health regulator said. Gilenya is called a sphingosine 1-phosphate receptor modulator, which is thought to act by retaining certain white blood cells in the lymph nodes, preventing them from crossing the blood-brain barrier into the central nervous system. Preventing the entry of these cells reduces inflammatory damage to the nervous system. Gilenya comes in capsules. It is taken by mouth once a day with or without food.
In a clinical trial 86 percent of patients receiving Gilenya remained relapse-free after 24 months of treatment, compared to 46 percent of those who were administered another MS drug.
Gilenya was first approved by the FDA to treat adults with relapsing MS. Some estimates suggest that up to five percent of cases of MS begin prior to adulthood. Pediatric MS generally manifests as active relapsing-remitting disease.
Potential Benefits for Children with Relapsing MS
This approval is based on phase 3 trial results, originally presented at the European Committee for Treatment and Research in MS in October 2017. Fingolimod reduced the annual number of relapses by 82% over two years, compared to treatment with interferon beta-1a (Avonex®, Biogen). After two years, 86% of those on fingolimod had not experienced a relapse, compared to 39% of the Avonex group. In MRI results reported at medical meetings, fingolimod significantly reduced disease activity on MRI scans, including the rate of brain volume loss.
Potential Risks for Children with Relapsing MS
Side effects seen in the phase 3 clinical trial in children and adolescents were similar to what have been seen in the adult population. The most common side effects were headache, liver enzyme elevation, diarrhea, cough, flu, sinusitis, back pain, abdominal pain and pain in extremities.
The prescribing information carries warnings about other potentially serious risks, including heart events on first dose, infections, an eye problem called macular edema, increases of liver enzymes (which could indicate liver injury) and progressive multifocal leukoencephalopathy (PML), a rare brain infection that can lead to death or severe disability. A medication guide will help inform patients and their families about its use and potential risks.