WASHINGTON, July 29, 2013 /PRNewswire/ -- Vanda
Pharmaceuticals Inc. (VANDA) (NASDAQ: VNDA) today announced that
the U.S. Food and Drug Administration (FDA) has accepted the filing
and granted a priority review classification to Vanda's New
Drug Application (NDA) for tasimelteon, a circadian regulator for
the treatment of Non-24-Hour Disorder (Non-24) in the totally
blind.
The FDA grants priority review status for a "drug that treats a
serious condition and, if approved, would provide a significant
improvement in safety or effectiveness" over current
therapies[1]. Currently, there is no approved
treatment for Non-24 and tasimelteon has the potential to address
this unmet medical need.
"We are extremely pleased that the FDA has granted tasimelteon
priority review for the treatment of Non-24 in the totally blind,"
said Mihael H. Polymeropoulos M.D.,
Vanda's President and Chief Executive Officer. "The agency's
acceptance of the NDA and decision to place tasimelteon in a
category of expedited review are important milestones for Vanda as
we take another step toward our goal of providing patients with a
treatment for Non-24."
The FDA determined the action target date under Prescription
Drug User Fee Act (PDUFA-V), to be January
31, 2014. The FDA has also tentatively scheduled an
advisory committee meeting to discuss the tasimelteon application
on November 14, 2013.
About Non-24-Hour Disorder
Non-24 is a serious, rare,
and chronic circadian rhythm disorder characterized by the
inability to entrain (synchronize) the master body clock with the
24-hour day-night cycle. Non-24 affects a majority of totally
blind individuals, or between 65,000 and 95,000 people in the
U.S. Non-24 occurs almost entirely in individuals who lack
the light sensitivity necessary to entrain the master body clock in
the brain with the 24-hour day-night cycle. Most people have
a master body clock that naturally runs longer than 24-hours and
light is the primary environmental cue that resets it to 24 hours
each day. Individuals with Non-24 have a master body clock
that is not reset, and continually delays, resulting in prolonged
periods of misalignment between their circadian rhythms and the
24-hour day-night cycle, including the timing of melatonin and
cortisol secretion. As a result of this misalignment, Non-24
is associated with significant disruption of the sleep-wake cycle
and impairments in social and occupational functioning, and marked
subjective distress. Currently there is no approved treatment
for Non-24. For more information on Non-24, please visit
www.Non-24.com.
About Tasimelteon
Tasimelteon is a circadian regulator
in development for the treatment of Non-24. Tasimelteon is a
dual melatonin receptor agonist (DMRA) with selective agonist
activity at the MT1 and MT2 receptors. Tasimelteon aims to
reset the master body clock in the suprachiasmatic nucleus (SCN),
resulting in the entrainment of the body's melatonin and cortisol
rhythms to align to the 24-hour day-night cycle. The patent
claiming tasimelteon as a new chemical entity extends through
December 2022, assuming a 5-year
extension to be granted under the Hatch-Waxman Act.
Tasimelteon has been granted orphan drug designation for the
treatment of Non-24 from both the U.S. and the European
Union. Tasimelteon has not been approved by the FDA or any
other regulatory authority.
About Tasimelteon NDA
Summary of
Efficacy
The efficacy of tasimelteon as a circadian
regulator of the master body clock in the treatment of Non-24 was
studied in two randomized, double-masked, placebo-controlled,
multicenter, parallel-group trials, SET (Safety and Efficacy of
Tasimelteon) and RESET (Randomized withdrawal Study of the Efficacy
and safety of Tasimelteon). In the SET study, tasimelteon
achieved the primary endpoints of entrainment (synchronizing) of
the melatonin rhythm and clinical response as measured by
entrainment plus an improvement on the Non-24 Clinical Response
Scale. Tasimelteon also demonstrated statistically
significant improvement in measures of nighttime sleep, daytime nap
duration, timing of sleep, and overall global functioning
scale. In patients treated with tasimelteon, daytime naps
decreased by 46 minutes per day in the worst 25% of days in a cycle
and nighttime sleep increased by 57 minutes per day during the
worst 25% of nights in a cycle. The RESET study demonstrated
that patients who continued treatment with 20 mg of tasimelteon
maintained entrainment of melatonin and cortisol circadian rhythms
at statistically significantly greater percentages than patients
receiving placebo. Patients treated with tasimelteon also
maintained their clinical benefits while patients who received
placebo showed significant deterioration in measures of nighttime
sleep, daytime naps and timing of sleep.
Summary of Safety
The Integrated Summary
of Safety (ISS) for the tasimelteon development program included
data from over 1,300 subjects treated with tasimelteon including
111 subjects treated for at least six months and 44 subjects
treated for at least one year. Common adverse events (>=2%
in tasimelteon and greater than placebo) in placebo controlled
studies (tasimelteon n=429, placebo n=203) included, (tasimelteon
%, placebo %), Back pain (2.1%, 2.0%), Dreams (vivid or unusual),
(2.6%, 0.5%), Diarrhea (2.3%, 1.0%), Dry Mouth (2.3%, 0.5%),
Headache (9.6%, 7.4%), Nasopharyngitis (6.5%, 6.4%), Somnolence
(3.0%, 1.5%), Upper Respiratory Tract Infection (2.6%, 1.5%).
In placebo controlled studies (tasimelteon n=429, placebo n=203),
all serious adverse events were deemed unrelated to study drug and
the rates between tasimelteon and placebo treated patients were
similar (1.6%, 1.5%).
About Vanda Pharmaceuticals Inc.
Vanda Pharmaceuticals
Inc. is a biopharmaceutical company focused on the development and
commercialization of products for the treatment of central nervous
system disorders. For more on Vanda, please visit
http://www.vandapharma.com.
Company Contact:
Jim
Kelly
Senior Vice President and Chief Financial Officer
Vanda Pharmaceuticals Inc.
(202) 734-3428
jim.kelly@vandapharma.com
Media Contact:
Laney Landsman
Assistant Vice President
Makovsky
(212) 508-9643
llandsman@makovsky.com
CAUTIONARY NOTE REGARDING FORWWARD LOOKING
STATEMENTS
Various statements in this release are
"forward-looking statements" under the securities laws. Words
such as, but not limited to, "believe," "expect," "anticipate,"
"estimate," "intend," "plan," "project," "target," "goal,"
"likely," "will," "would," and "could," or the negative of these
terms and similar expressions or words, identify forward-looking
statements. Forward-looking statements are based upon current
expectations that involve risks, changes in circumstances,
assumptions and uncertainties. Important factors that could
cause actual results to differ materially from those reflected in
the company's forward-looking statements include, among others:
Vanda's failure to obtain, or any delay in obtaining, regulatory
approval for tasimelteon for the treatment of Non-24-Hour Disorder
or to comply with ongoing regulatory requirements and other factors
that are described in the "Risk Factors" and "Management's
Discussion and Analysis of Financial Condition and Results of
Operations" sections of Vanda's annual report on Form 10-K for the
fiscal year ended December 31, 2012
which is on file with the SEC and available on the SEC's website at
www.sec.gov. In addition to the risks described above and in
Vanda's annual report on Form 10-K and quarterly reports on Form
10-Q, other unknown or unpredictable factors also could affect
Vanda's results. There can be no assurance that the actual
results or developments anticipated by Vanda will be realized or,
even if substantially realized, that they will have the expected
consequences to, or effects on, Vanda. Therefore, no
assurance can be given that the outcomes stated in such
forward-looking statements and estimates will be achieved.
All written and verbal forward-looking statements attributable
to Vanda or any person acting on its behalf are expressly qualified
in their entirety by the cautionary statements contained or
referred to herein. Vanda cautions investors not to rely too
heavily on the forward-looking statements Vanda makes or that are
made on its behalf. The information in this release is
provided only as of the date of this release, and Vanda undertakes
no obligation, and specifically declines any obligation, to update
or revise publicly any forward-looking statements, whether as a
result of new information, future events or otherwise.
[1] U.S. Department of Health and Human Services Food
and Drug Administration, "Draft Guidance for Industry: Expedited
Programs for Serious Conditions—Drugs and Biologics" June 2013.
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf
[Last accessed June 22, 2013]
SOURCE Vanda Pharmaceuticals Inc.