WASHINGTON, Dec. 1, 2020 /PRNewswire/ -- Vanda
Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that
the U.S. Food and Drug Administration (FDA) has approved
HETLIOZ® (tasimelteon) capsule and liquid formulations
for the treatment of adults and children, respectively, with
nighttime sleep disturbances associated with Smith-Magenis Syndrome
(SMS). SMS is a rare neurodevelopmental disorder, a defining
feature of which is an "inverted" circadian rhythm, making it
extremely difficult for patients with SMS to sleep during the
night. HETLIOZ® is the first FDA-approved medication for
patients with SMS.
"The FDA approval of HETLIOZ® for the treatment of
nighttime sleep disturbances in SMS would not have been
accomplished without the heroic efforts of SMS patients and the
efforts of their families and advocates supporting the recruitment,
design, and conduct of the study," said Mihael H. Polymeropoulos M.D., Vanda's President
and CEO. "We remain committed to providing this much needed therapy
to patients with SMS."
The approval of HETLIOZ® for the treatment of
nighttime sleep disturbances in SMS was based on a single
placebo-controlled efficacy study in this rare disorder, which
studied both adults with SMS taking the HETLIOZ® capsule
and children with SMS taking the liquid formulation. The safety
profile of HETLIOZ® in this study was similar to those
seen in HETLIOZ® studies previously conducted for the
treatment of Non-24-Hour Sleep-Wake Disorder, and was similar
between adults and children with SMS.
"We are very excited to see HETLIOZ®, the first ever
treatment approved for people with SMS, addressing the significant
problem of sleep disturbances and we are happy to see this
treatment used in our community," said Maggie Miller, Co-Founder and Vice President of
PRISMS (Parents and Researchers Interested in Smith-Magenis
Syndrome). "We thank Vanda and the FDA for partnering to help
our community and we look forward to continued partnership to bring
this important therapy to families with SMS."
HETLIOZ® capsules, for adults with SMS, will be
immediately available and the HETLIOZ LQ™ liquid
formulation, for children with SMS, is expected to be available in
the first quarter of 2021.
About Smith-Magenis Syndrome
Smith-Magenis Syndrome (SMS) is a developmental disorder that is
caused by a small deletion of human chromosome
17p 1,2. In more rare cases SMS is caused by a
point mutation in the RAI1 gene which resides in the deleted
region. SMS is estimated to affect 1/15,000-25,000 births in
the U.S.3 SMS is usually not inherited but rather
is due to a de-novo deletion. Patients with SMS present with a
number of physical, mental and behavioral problems. The most common
symptom of SMS is a severe sleep disorder associated with
significant disruption in the lives of patients and their
families.
References:
- Williams, S. R., Zies, D., Mullegama, S. V, Grotewiel, M. S.,
& Elsea, S. H. (2012). Smith-Magenis syndrome results in
disruption of CLOCK gene transcription and reveals an integral role
for RAI1 in the maintenance of circadian rhythmicity. Am.J
Hum.Genet., 90(1537–6605), 941–949.
- Gropman, A. L., Duncan, W. C., & Smith, A. C. (2006).
Neurologic and developmental features of the Smith-Magenis syndrome
(del 17p11.2). Pediatr.Neurol., 34(0887–8994), 337–350.
- Orphanet ORPHA number 819.
About Vanda Pharmaceuticals Inc.
Vanda is a leading global biopharmaceutical company focused on
the development and commercialization of innovative therapies to
address high unmet medical needs and improve the lives of patients.
For more on Vanda Pharmaceuticals Inc., please visit
www.vandapharma.com and follow us on Twitter @vandapharma.
About HETLIOZ®
HETLIOZ® (tasimelteon) is a melatonin receptor
agonist. HETLIOZ® has been granted market authorization
by the U.S. Food and Drug Administration and the European Medicines
Agency. For full U.S. Prescribing Information for
HETLIOZ®, including indication and Important Safety
Information, visit www.hetlioz.com.
CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS
Various statements in this release, including, but not limited
to statements regarding Vanda's ability to make HETLIOZ®
available to patients with nighttime sleep disturbances in SMS, are
"forward-looking statements" under the securities laws.
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 Vanda's forward-looking
statements include, among others, market acceptance of
HETLIOZ® as a treatment of SMS in adults and children,
Vanda's dependence on third-party manufacturers to manufacture
HETLIOZ® in sufficient quantities and quality, and
Vanda's sales and marketing infrastructure. 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 will be achieved. Forward-looking
statements in this press release should be evaluated together with
the various risks and uncertainties that affect Vanda's business
and market, particularly those identified in the "Cautionary Note
Regarding Forward-Looking Statements", "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,
2019, as updated by Vanda's subsequent Quarterly Reports on
Form 10-Q, Current Reports on Form 8-K and other filings with the
U.S. Securities and Exchange Commission, which are available at
www.sec.gov.
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 press release is
provided only as of the date of this press 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,
except as required by law.
Corporate Contact:
AJ Jones II
Chief Corporate Affairs and Communications Officer
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
Elizabeth Van Every
Head of Corporate Affairs
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
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SOURCE Vanda Pharmaceuticals Inc.