Gilenya® (fingolimod, FTY720)
Novartis Pharmaceuticals Corp.
Study Information (we refer you to our 2010
Research Update for more details):
- Oral medication; 0.5 mg capsule taken once
- 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
- 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.
- 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
- 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.