WASHINGTON, Dec. 4, 2023
/PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (NASDAQ: VNDA)
today announced that the U.S. Food and Drug Administration (FDA)
has accepted the filing of Vanda's New Drug Application (NDA) for
tradipitant for the treatment of symptoms of gastroparesis.
The FDA has set September 18, 2024
as the target date for its decision under the Prescription Drug
User Fee Act (PDUFA). If approved, tradipitant will be the first
novel drug to be approved by the FDA for the treatment of
gastroparesis in over 40 years and to be accepted for review by the
FDA for gastroparesis in over 30 years.
Gastroparesis is a serious medical condition characterized by
delayed gastric emptying associated with the symptoms of nausea,
vomiting, bloating, fullness after meals and abdominal pain, along
with significant impairment of social and occupational functioning.
The estimated prevalence of gastroparesis in the U.S. is
approximately 6 million patients, many of whom remain
undiagnosed.1
In support of this application, the FDA accepted for review
Vanda's non-animal preclinical toxicology data derived from
microphysiological systems that was provided by Vanda in the
absence of a nine-month toxicity dog study. Vanda believes that
this is an important milestone in its multi-year effort to convince
the FDA to move away from animal toxicology studies and instead
adopt novel and advanced human-relevant technologies.
"We are very pleased with the FDA's acknowledgment of the
completeness of our application and we look forward to a
substantive review. Tradipitant, if approved, will be the first
novel drug for patients with gastroparesis since 1979," said
Mihael H. Polymeropoulos, M.D.,
Vanda's President, CEO and Chairman of the Board. "We are also
encouraged by the FDA's decision to accept for filing this
application that includes non-animal preclinical toxicity data from
advanced human-relevant microphysiological systems in the absence
of a nine-month dog study."
The NDA submission includes results from clinical efficacy
studies 2301 and 3301, evidence from a large 12-week open label
study and data from the Expanded Access program.
The safety database in this application now under review
includes over 1,000 patients with exposures of up to 12-weeks, as
well as data from the Expanded Access program with patient
exposures of over two years.
The FDA's action on this application will mark the third NDA or
supplemental New Drug Application (sNDA) decision expected by Vanda
in 2024; a sNDA for HETLIOZ® for the treatment of
insomnia characterized by difficulties with sleep initiation has a
PDUFA target action date of March 4,
2024 and a sNDA for Fanapt® for the acute
treatment of manic or mixed episodes associated with bipolar I
disorder has a PDUFA target action date of April 2, 2024.
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 X @vandapharma.
About Tradipitant
Tradipitant is an NK-1R antagonist licensed by Vanda from Eli
Lilly and Company. Tradipitant is currently in clinical development
for gastroparesis and motion sickness. The FDA has imposed a
partial clinical hold on tradipitant clinical protocols of longer
than 12 weeks duration.
About Gastroparesis
Gastroparesis is a serious medical condition characterized by
delayed gastric emptying associated with the symptoms of nausea,
vomiting, bloating, fullness after meals and abdominal pain, along
with significant impairment of social and occupational functioning.
The estimated prevalence of gastroparesis in the U.S. is
approximately 6 million patients, many of whom remain
undiagnosed.1 Gastroparesis affects mostly women and it
can be of diabetic, idiopathic or other etiology. The only FDA
approved treatment for gastroparesis is metoclopramide, approved in
1979, which due to its potential of severe side effects carries a
black box warning and limitations of use of no more than 3 months.
Patients are faced with limited therapeutic options and clinical
guidelines recommend, in addition to metoclopramide, the off label
use of different drugs including erythromycin, domperidone (not
approved in the U.S.), botulinum toxin injections, gastric
stimulators and a variety of surgical procedures in an effort to
relieve even temporarily some of the symptoms of the
disease.2 Gastroparesis treatment represents a
significant unmet medical need as underscored by the testimonies of
interested parties and advocacy organizations including the
International Foundation for Gastrointestinal Disorders (IFFGD) and
Gastroparesis Patient Association for Cures and Treatments, Inc.
(G-Pact).
References
- Rey, E., Choung, R. S., Schleck, C. D., Zinsmeister, A. R.,
Talley, N. J., & Locke, G. R., 3rd (2012). Prevalence of hidden
gastroparesis in the community: the gastroparesis
"iceberg". Journal of neurogastroenterology and
motility, 18(1), 34–42.
https://doi.org/10.5056/jnm.2012.18.1.34
- Camilleri, M., Chedid, V., Ford, A. C., Haruma, K., Horowitz,
M., Jones, K. L., Low, P. A., Park, S. Y., Parkman, H. P., &
Stanghellini, V. (2018). Gastroparesis. Nature reviews.
Disease primers, 4(1), 41.
https://doi.org/10.1038/s41572-018-0038-z
CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS
Various statements in this press release, including, but not
limited to, statements regarding the FDA's review of the NDA for
tradipitant and the potential approval of tradipitant for the
treatment of symptoms of gastroparesis, the prevalence of
gastroparesis in the U.S., the FDA's acceptance for filing of the
NDA for tradipitant in the absence of data from a nine-month
toxicity dog study and Vanda's expectations regarding the timing of
the FDA's decisions with respect to the NDA for tradipitant and the
sNDAs for HETLIOZ® and Fanapt® are
"forward-looking statements" under the securities laws. All
statements other than statements of historical fact are statements
that could be deemed forward-looking statements. Forward-looking
statements are based upon current expectations and assumptions that
involve risks, changes in circumstances 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 FDA's assessment of the
adequacy of the data included in the NDA for tradipitant and its
willingness to approve the NDA without data from a nine-month
toxicity dog study, the accuracy of the estimates of the number of
patients in the U.S. with gastroparesis, the FDA's assessment of
the data included in the sNDA's for HETLIOZ® and
Fanapt® and the FDA's ability to meet the PDUFA target
action dates for the NDA for tradipitant and the sNDAs for
HETLIOZ® and Fanapt®. Therefore, no assurance
can be given that the 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.
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
most recent Annual Report on Form 10-K, 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:
Kevin
Moran
Senior Vice President, Chief Financial Officer and Treasurer
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
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SOURCE Vanda Pharmaceuticals Inc.