WASHINGTON, May 13, 2020 /PRNewswire/ -- Vanda
Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that
following the completion of a Type A Meeting with the U.S. Food and
Drug Administration (FDA) on May 8,
2020, it has reached agreement with the FDA to resubmit its
application for HETLIOZ® (tasimelteon) for the treatment of
Smith-Magenis Syndrome (SMS). As previously disclosed, Vanda
received a Refusal to File letter from the FDA on March 12, 2020. The Type A Meeting resolved
the outstanding issues regarding the filing of the
application. Vanda plans to resubmit as soon as possible,
seeking approval of the solid capsule formulation of HETLIOZ® for
the treatment of adults with SMS, and the liquid formulation of
HETLIOZ ® for the treatment of children with SMS*.
"We are very pleased with the outcome of the Type A Meeting,"
said Dr. Mihael H. Polymeropoulos,
President and CEO of Vanda. "This meeting was a great example of
collaboration with the agency and we appreciate FDA's thoughtful
work in helping us advance the progress of our application and
bring this potentially important treatment closer to use by
patients with SMS."
In December 2018, Vanda reported
results of the largest placebo controlled study ever conducted in
patients with SMS, with HETLIOZ® patients seeing significant
improvements in sleep. SMS is a developmental disorder that
is frequently caused by a small deletion of human chromosome
17p.1, 2 In some cases, SMS is caused by a point
mutation in the RAI1 gene, which resides in the deleted region. SMS
is estimated to affect 1 in 15,000-25,000 individuals.3
Patients with SMS present with a number of physical, mental
and behavioral issues. The most common symptom of SMS is a
severe sleep disorder, which results in significant disruption in
the lives of patients and their families.
*Vanda's resubmission will be in the form of a supplemental New
Drug Application for the solid capsule formulation of HETLIOZ® for
the treatment of adults with SMS (the sNDA) and a New Drug
Application for the liquid formulation of HETLIOZ ® for the
treatment of children with SMS (the NDA). Originally, Vanda
submitted an sNDA covering both formulations, but the FDA requested
that Vanda separate that application into an sNDA and an NDA for
the different formulations.
References:
1 Williams, SR, Zies, D, Mullegama, SV, Grotewiel,
MS, & Elsea, SH. 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. 2012;
90(1537–6605), 941–949.
2 Gropman, AL, Duncan, WC, & Smith, AC.
Neurologic and developmental features of the Smith-Magenis syndrome
(del 17p11.2). Pediatr Neurol. 2006; 34(0887–8994), 337–350.
3 Greenberg F, Guzzetta V, Montes de Oca-Luna R et
al: Molecular analysis of the Smith –Magenis syndrome: a possible
contiguous gene syndrome associated with del(17)(p11.2). Am J Hum
Genet. 1991; 49:1207 – 1218.
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 Vanda's Twitter and
LinkedIn.
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
CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS
Various statements in this release, including, but not limited
to statements regarding Vanda's HETLIOZ® program for the treatment
of SMS and plans to resubmit the sNDA and the NDA related to such
program, 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, Vanda's ability
to complete the preparation of and submit the sNDA and the NDA; the
FDA's acceptance and review of such filings; Vanda's ability to
obtain FDA approval of HETLIOZ® for the treatment of SMS in adults
and children; 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, 2019 and quarterly report on Form
10-Q for the quarter ended March 31,
2020, which are 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.
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SOURCE Vanda Pharmaceuticals Inc.