FDA Accepts Tasimelteon New Drug Application For Priority Review In The Treatment Of Non-24-Hour Disorder In The Totally Blind

   FDA Accepts Tasimelteon New Drug Application For Priority Review In The
            Treatment Of Non-24-Hour Disorder In The Totally Blind

PR Newswire

WASHINGTON, July 29, 2013

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
toVanda'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.

Website: http://www.vandapharma.com