WASHINGTON, Aug. 3, 2020 /PRNewswire/ -- Vanda
Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that
the U.S. Food and Drug Administration (FDA) has accepted for
priority review Vanda's applications for Smith-Magenis Syndrome
(SMS). The applications include a Supplemental New Drug Application
(sNDA) for HETLIOZ® capsules and a New Drug Application
(NDA) for the liquid formulation of HETLIOZ® for
the treatment of adults and children, respectively, with
Smith-Magenis Syndrome (SMS). The FDA has set December 1, 2020 as the target date for its
decision under the Prescription Drug User Fee Act (PDUFA-VI).
"The FDA filing of the HETLIOZ® applications for
priority review marks a major milestone and brings us closer to
providing a critical therapy to patients with SMS," said
Mihael H. Polymeropoulos, M.D.,
Vanda's President and CEO. Currently, there are no approved
treatments for patients with SMS, a rare orphan disorder affecting
approximately 15,000 people in the U.S.
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.
HETLIOZ® IS NOT CURRENTLY APPROVED BY ANY
REGULATORY AUTHORITY FOR THE TREATMENT OF SMS.
CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS
Various statements in this release, including, but not limited
to statements regarding the target completion date of the FDA's
review of the sNDA and NDA for HETLIOZ® and Vanda's
ability to make HETLIOZ® available to patients with 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, the ability of the FDA to
complete its review of the applications on time and make the
determination that HETLIOZ® is safe and effective in the
treatment of SMS in adults and children. 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.