Gilenya (fingolimod), manufactured by Novartis, is a treatment approved by the U.S. Food and Drug Administration (FDA) for patients with relapsing-remitting multiple sclerosis (RRMS) starting at age 10.
This oral disease-modifying therapy modulates the immune response and works to reduce disease exacerbations, so as to delay progression and disability accumulation.
How Gilenya works
In multiple sclerosis (MS), the immune system mistakenly targets healthy myelin, the main component of the insulating and protective sheath that surrounds nerve fibers. Immune cells, or lymphocytes, can freely cross the blood-brain barrier and cause inflammation and damage to the central nervous system (CNS), resulting in progressive nerve cell death (neurodegeneration).
Gilenya is a type of sphingosine 1-phosphate (S1P) receptor modulator. S1P receptors are present on the outside of the lymphocytes and have various roles, including regulating the lymphocytes leaving the lymph nodes, where they are made and stored.
By binding to the S1P receptor, Gilenya acts to retain lymphocytes in the lymph nodes and prevent them from reaching the CNS. This should lower the number of lymphocytes in the brain, reducing inflammation and CNS damage.
S1P receptors are also found on some cells of the central nervous system, and by binding to these cells, Gilenya may promote their survival and growth, potentially stimulating re-myelination.
Gilenya in clinical trials
Gilenya was approved by the FDA in September 2010 to treat RRMS in patients ages 18 and older, based on the results of three key Phase 3 clinical trials: FREEDOMS (NCT00289978), FREEDOMS II (NCT00355134), and TRANSFORMS (NCT00340834).
FREEDOMS enrolled 1,272 RRMS patients, ages 18 to 55, at trial sites worldwide. Over a two-year period, patients were given Gilenya at a low or high dose, or a placebo, daily. Results, published in the New England Journal of Medicine, demonstrated that Gilenya significantly reduced the number of relapses and slowed disability progression.
The TRANSFORMS study compared Gilenya to another established MS therapy, Avonex (interferon beta-1a), over the course of one year. A total of 1,153 RRMS patients who had recently relapsed were enrolled at trial sites worldwide. The results, published in the New England Journal of Medicine, suggested that Gilenya was more effective than Avonex in reducing the number of relapses, supported by brain imaging scans showing fewer lesions. No significant difference in the progression of disability was seen between the two treatments.
Extension trials have shown that this lowering in disease activity could be maintained for at least seven years.
After observing its safety and efficacy in extensive clinical trials, Gilenya became the first oral treatment for relapsing-remitting forms of MS with its FDA approval in September 2010. Health Canada and the European Medicines Agency (EMA) also approved the treatment for these patients in March 2011.
On May 11, 2018, the FDA extended Gilenya’s approval to include pediatric RRMS patients, ages 10 or older, based on the results of a Phase 3 clinical trial (NCT01892722) called PARADIGMS. The trial enrolled 215 children with relapsing MS, ages 10 to 18, and randomized them to receive either Gilenya or Avonex for up to two years. Results, published in The New England Journal of Medicine, demonstrated that Gilenya was associated with a lower relapse rate and fewer additional brain lesions as detected by magnetic resonance imaging (MRI). However, a high rate of serious adverse events was reported with treatment use, including infection in four patients and leukopenia, or a low white blood cell count in two patients.
The Committee for Medicinal Products for Human Use (CHMP) also supported Gilenya as a RRMS pediatric treatment in a favorable opinion to the EMA in September 2018. A decision regarding its use in young RRMS patients in Europe is expected by year’s end.
Common side effects associated with Gilenya include dizziness, headaches, nausea, and fatigue. Since the treatment modulates the immune system, patients with infectious diseases should avoid using it, as these infections may worsen as a result of a temporarily less active immune system.
The FDA announced in May 2012 that Gilenya is contraindicated in patients with certain pre-existing or recent heart conditions or stroke, or who are taking certain anti-arrhythmic medications. Pregnant women are also advised not to take Gilenya.
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