Aquestive Therapeutics, Inc. (NASDAQ: AQST), a pharmaceutical
company focused on developing and commercializing differentiated
products that address patients’ unmet needs and solve therapeutic
problems, announced today that, regarding the review of the New
Drug Application (NDA) for Libervant™ (diazepam) Buccal Film for
the treatment of breakthrough seizures and seizure clusters, the
U.S. Food & Drug Administration (FDA) sent the following notice
to the Company;
“Please refer to your
NDA 212641 Libervant resubmission dated June 23, 2021.
The purpose of this
e-mail is to notify you that the Agency is still considering the
regulatory issues related to the approvability of Libervant, and
will not be ready to take an action by December 23, 2021. No
additional information is needed from you at this time. We are
currently unable to provide an estimate of the timing of an
expected action; however, we will provide any updates we can as
they are available.”
“We are surprised to receive this notification
from the FDA. We accepted all of the Agency’s label edits for
Libervant last week and, as the Agency stated in its notice today,
there is no additional information needed from the Company at this
time. Following the labeling discussions, we believe the package we
submitted satisfies all of CDER’s requests,” said Keith Kendall,
Chief Executive Officer of Aquestive. “We are currently reaching
out to the FDA to seek further clarity. We remain fully committed
to working closely with the FDA for the duration of its review of
our NDA. We continue to believe that Libervant provides a major
contribution to patient care and has the potential to transform the
lives of refractory epilepsy patients seeking a non-invasive and
innovative product for the management of seizure clusters.”
In July 2021, the FDA accepted the NDA for
Libervant and assigned a Prescription Drug User Fee Act (“PDUFA”)
target goal date of December 23, 2021.
The Company continues to advance AQST-109 as it began the
EPIPHAST study, the results of which are expected to be available
in the first half of 2022.
About LibervantLibervant™ is a
buccally, or inside of the cheek, administered soluble film
formulation of diazepam, a benzodiazepine intended for the acute
treatment of intermittent, stereotypic episodes of frequent seizure
activity (i.e., seizure clusters, acute repetitive seizures) that
are distinct from a patient’s usual seizure pattern in patients
with epilepsy 12 years of age and older. The Company believes that
Libervant will enable a larger share of patients to receive more
appropriate treatment by providing consistent therapeutic dosing in
a non-invasive and innovative treatment form for epileptic
seizures. Aquestive developed Libervant as an alternative to more
invasive, inconvenient, and difficult to administer device driven
products, including a rectal gel and nasal spray, for patients with
refractory epilepsy. A large portion of the patient population
still does not access either one of these existing products and
remains underserved with regard to having an important medication
where they need it, when they need it and in a form they prefer.
Libervant was granted Orphan Drug Designation by the FDA in
November 2016.
About AQST-109AQST-109 is a
polymer matrix-based film that can be applied sublingually (under
the tongue) for the rapid delivery of epinephrine. The product is
similar in size to a postage stamp, weighs less than an ounce, and
begins to dissolve on contact. No water or swallowing is required
for administration. The packaging for AQST-109 is thinner and
smaller than an average credit card, can be carried in a pocket,
and is designed to withstand weather excursions such as exposure to
rain and/or sunlight. The Company has begun recruiting subjects for
its EPIPHAST adaptive design study. Results of the study will be
available in the first half of 2022.
About AnaphylaxisAnaphylaxis is
a potentially life-threatening systemic allergic reaction, with an
estimated incidence of 50 to 112 episodes per 100,000 people per
year. The frequency of hospital admissions for anaphylaxis has
increased 500-700% in the last 10-15 years. The most common causes
of reactions that can include anaphylaxis are medications, foods
(such as peanuts), and venom from insect stings. Epinephrine
injection is the current standard of treatment intended to reverse
the potentially severe manifestation of anaphylaxis, which may
include red rash, throat swelling, respiratory problems,
gastrointestinal distress and loss of consciousness.
About Aquestive
TherapeuticsAquestive Therapeutics is a pharmaceutical
company that applies innovative technology to solve therapeutic
problems and improve medicines for patients. The Company has
commercialized one internally-developed proprietary product to
date, Sympazan® (clobazam) oral film, has a commercial proprietary
product pipeline focused on the treatment of diseases of the
central nervous system, or CNS, and other unmet needs, and is
developing orally administered complex molecules to provide
alternatives to invasively administered standard of care therapies.
The Company also collaborates with other pharmaceutical companies
to bring new molecules to market using proprietary, best-in-class
technologies, like PharmFilm®, and has proven capabilities for drug
development and commercialization.
Forward-Looking
StatementCertain statements in this press release include
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995. Words such as “believe,”
“anticipate,” “plan,” “expect,” “estimate,” “intend,” “may,”
“will,” or the negative of those terms, and similar expressions,
are intended to identify forward-looking statements. These
forward-looking statements include, but are not limited to,
statements regarding the advancement and related timing of
Libervant through FDA approval, the advancement and related timing
of AQST-109 through the development pipeline and regulatory
approval, and clinical and business strategies, market
opportunities, and other statements that are not historical facts.
These forward-looking statements are subject to the uncertain
impact of the COVID-19 global pandemic on our business including
with respect to our clinical trials including site initiation,
patient enrollment and timing and adequacy of clinical trials; on
regulatory submissions and regulatory reviews and approvals of our
product candidates; pharmaceutical ingredient and other raw
materials supply chain, manufacture, and distribution; sale of and
demand for our products; our liquidity and availability of capital
resources; customer demand for our products and services;
customers’ ability to pay for goods and services; and ongoing
availability of an appropriate labor force and skilled
professionals. Given these uncertainties, the Company is unable to
provide assurance that operations can be maintained as planned
prior to the COVID-19 pandemic.
These forward-looking statements are based on
our current expectations and beliefs and are subject to a number of
risks and uncertainties that could cause actual results to differ
materially from those described in the forward-looking statements.
Such risks and uncertainties include, but are not limited to, risks
associated with the Company’s development work, including any
delays or changes to the timing, cost and success of our product
development activities and clinical trials for AQST-109 and our
other product candidates; risk of delays in FDA approval of
AQST-109, Libervant and our other drug candidates or failure to
receive FDA approval for any of our product candidates; ability to
address the concerns identified in the FDA’s Complete Response
Letter dated September 25, 2020 regarding the New Drug Application
for Libervant; risk of our ability to demonstrate to the FDA
“clinical superiority” within the meaning of the FDA regulations of
Libervant relative to FDA-approved diazepam rectal gel and nasal
spray products including by establishing a major contribution to
patient care within the meaning of FDA regulations relative to the
approved products as well as risks related to other potential
pathways or positions which are or may in the future be advanced to
the FDA to overcome the seven year orphan drug exclusivity granted
by the FDA for the approved nasal spray product of a competitor in
the U.S. and there can be no assurance that we will be successful;
risk that a competitor obtains FDA orphan drug exclusivity for a
product with the same active moiety as any of our other drug
products for which we are seeking FDA approval and that such
earlier approved competitor orphan drug blocks such other product
candidates in the U.S. for seven years for the same indication;
risk in obtaining market access for other reasons; risk inherent in
commercializing a new product (including technology risks,
financial risks, market risks and implementation risks and
regulatory limitations); risk of development of our sales and
marketing capabilities; risk of legal costs associated with and the
outcome of our patent litigation challenging third party at risk
generic sale of our proprietary products; risk of sufficient
capital and cash resources, including access to available debt and
equity financing and revenues from operations, to satisfy all of
our short-term and longer term liquidity and cash requirements and
other cash needs, at the times and in the amounts needed; risks
related to the outsourcing of certain marketing and other
operational and staff functions to third parties; risk of the rate
and degree of market acceptance of our product and product
candidates; the success of any competing products, including
generics; risk of the size and growth of our product markets; risks
of compliance with all FDA and other governmental and customer
requirements for our manufacturing facilities; risks associated
with intellectual property rights and infringement claims relating
to the Company’s products; risk of unexpected patent developments;
the impact of existing and future legislation and regulatory
provisions on product exclusivity; legislation or regulatory
actions affecting pharmaceutical product pricing, reimbursement or
access; claims and risks that may arise regarding the safety or
efficacy of the Company's products and product candidates; risk of
loss of significant customers; risks related to legal proceedings,
including patent infringement, investigative and antitrust
litigation matters; changes in government laws and regulations;
risk of product recalls and withdrawals; uncertainties related to
general economic, political, business, industry, regulatory and
market conditions and other unusual items; and other uncertainties
affecting the Company described in the “Risk Factors” section and
in other sections included in our Annual Report on Form 10 K, in
our Quarterly Reports on Form 10-Q, and in our Current Reports on
Form 8-K filed with the Securities Exchange Commission. Given those
uncertainties, you should not place undue reliance on these
forward-looking statements, which speak only as of the date made.
All subsequent forward-looking statements attributable to us or any
person acting on our behalf are expressly qualified in their
entirety by this cautionary statement. The Company assumes no
obligation to update forward-looking statements or outlook or
guidance after the date of this press release whether as a result
of new information, future events or otherwise, except as may be
required by applicable law.
PharmFilm®, Sympazan® and the Aquestive logo are
registered trademarks of Aquestive Therapeutics, Inc.
Investor InquiriesWestwicke, an ICR
CompanyStephanie
CarringtonStephanie.carrington@westwicke.com646-277-1282
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