Gilenya® (fingolimod, FTY720)

Parent company: Novartis Pharmaceuticals Corp.

• Oral medication; 0.5 mg capsule taken once daily.
  
  
• Approved for relapsing forms of MS.
  
  
• Gilenya (pronounced as "Jil-EN-ee-ah") is the first in a new class of immunomodulatory drugs, called "S1P-receptor modulators." It is similar in structure to a naturally occurring component of cell-surface receptors on white blood cells. (White blood cells are produced by the immune system to fight infection and disease.) Gilenya blocks potentially damaging T cells from leaving lymph nodes, lowering their number in the blood and tissues. It may reduce damage to the central nervous system (CNS) and enhance the repair of damaged nerves. The CNS consists of the brain, optic nerves, and spinal cord. Study data suggest that Gilenya may have neuroprotective effects.
  
  
• Some adverse events with Gilenya include: an initial reduction in heart rate; infrequent transient AV conduction block of the heart; macular edema (a condition that can affect vision, caused by swelling behind the eye); and infections, including herpes.

Study Information (we refer you to our 2010 Research Update for more details):

• The FREEDOMS Phase III study of the (now FDA-approved) low-dose (0.5 mg) versus highdose (1.25 mg) Gilenya and placebo showed the drug to be safe and well tolerated. Gilenya reduced the risk of confirmed disability progression by 30 to 32 percent versus placebo, and significantly increased the proportion of patients who were disease-free over two years. It also resulted in a 30-percent reduction of brain volume loss as compared with placebo at one and two years, suggesting a possible direct neuroprotective effect.
  
  
• A Phase III extension study, FREEDOMS II, evaluated long-term safety, tolerability, and efficacy; all 1,080 participants received Gilenya. Its safety has now been tested in more than 2,600 patients. More than 10,000 patients in the United States have begun therapy with Gilenya.
  
  
• Two deaths from herpes virus infections occurred in the FREEDOMS trials; both of these individuals received a higher dose of Gilenya, which is not FDA-approved or prescribed. No deaths were reported in those individuals treated with the FDA-approved lower dose, the only dose available for MS patients.
  
  
• The TRANSFORMS Phase III trial was a 12- month study of the efficacy of two doses of Gilenya (0.5 mg and 1.25 mg) as compared to weekly intramuscular injections of Avonex in individuals with RRMS. In summary, Gilenya was more effective in reducing the annual relapse rate, resulted in less deterioration in the ability to independently perform daily activities, was associated with a lower rate of brain atrophy, and showed a greater effect on reducing MRI measures of lesion activity. No difference in progression of disability was demonstrated in this 12-month study.
  
  
• In both the FREEDOMS and TRANSFORMS studies, Gilenya significantly reduced the frequency of severe relapses and those that required intervention (steroids or hospitalization), and reduced the number of relapses with no or partial recovery. It also consistently reduced the annualized relapse rate in patients with highly active MS as compared to Avonex.
  
  
• The 36-month INFORMS study in 940 individuals will evaluate the effect of Gilenya relative to placebo on delaying the time to sustained disability progression in patients with PPMS. It will also evaluate safety, tolerability, and the effects on MRI parameters.
  
  
• The six-month Phase IV EPOC study is currently recruiting participants. It will evaluate patientreported outcomes, physician assessment of a change, as well as safety and tolerability in patients with relapsing MS who had previously been treated with other DMTs and are now receiving Gilenya. These outcomes will be compared to those who continue to receive one of the other approved DMTs. The study will have approximately 1,000 participants and is scheduled for completion in June 2012.
  
  
• A long-term study of approximately 1,200 people with the relapsing forms of MS began in February 2011 and is scheduled to terminate in February 2019. The study will collect long-term data on safety and effectiveness.