Aquestive Therapeutics, Inc. (NASDAQ: AQST), a specialty
pharmaceutical company focused on developing and commercializing
differentiated products that address patients’ unmet needs and
solve therapeutic problems, announced today that, as anticipated,
the U.S. Food and Drug Administration (FDA) accepted the Company’s
New Drug Application (NDA) for Libervant™ (diazepam) Buccal Film
for the management of seizure clusters. The FDA has assigned a
Prescription Drug User Fee Act (PDUFA) goal date of September 27,
2020. If approved by the FDA, Libervant will be the first
oral diazepam-based therapy approved for management of seizure
clusters in the population of 1.2 million refractory epilepsy
patients. Libervant was designated by the FDA as an orphan
drug in November 2016.
“The FDA filing acceptance for Libervant is an
important milestone in our mission to provide epilepsy patients
with a broader array of treatment options, that represent major
contributions to patient care,” said Keith J. Kendall, Chief
Executive Officer of Aquestive. “Aquestive is committed to helping
people affected by seizure clusters through bringing important and
innovative products to the market. Epilepsy patients have
been underserved for some time with little choice beyond
device-based products such as rectally administered gels and nasal
sprays. We believe that our drug candidate Libervant
will, if approved by the FDA, represent a major contribution
to patient care, as compared to available treatment options, and
further expand patient choice as the first orally administered
dosage form available to manage seizure clusters in epilepsy
patients.”
“The FDA has recently indicated that, when
evaluating clinical superiority for drugs demonstrating a ‘major
contribution to patient care,’ it may consider such factors as
convenience of treatment location, duration of treatment, patient
comfort, reduced treatment burden, advances in ease and comfort of
drug administration, longer periods between doses, and potential
for self-administration,” continued Mr. Kendall. “We look
forward to working with the FDA in the coming months in seeking to
demonstrate why we believe that our product candidate Libervant, as
an orally delivered product for this indication, has one or more of
the attributes required by the FDA to be considered a major
contribution to patient care relative to the currently approved
products.”
About LibervantLibervant™ is a
buccally, or inside of the cheek, administered soluble film
formulation of diazepam, a benzodiazepine intended for rapid
treatment of acute uncontrolled seizures in selected, refractory
patients with epilepsy on stable regimens of AEDs who require
intermittent use of diazepam to control bouts of increased seizure
activity. Aquestive is developing Libervant as an alternative to
Diastat (diazepam rectal gel), the current standard of care rescue
therapy for patients with refractory epilepsy, which as a rectal
gel, is invasive, inconvenient, and difficult to administer. As a
result, a large portion of the patient population does not receive
adequate treatment or foregoes treatment altogether. It is
anticipated that Libervant will enable a larger share of these
patients to receive more appropriate treatment by providing
consistent therapeutic dosing in a non-invasive and innovative
treatment form for epileptic seizures.
The NDA submission was supported by data from
Aquestive’s single dose crossover study that was recently featured
as a late breaker session at the American Epilepsy Society (AES)
2019 Annual Meeting and expanded upon by key opinion leaders at its
investor and analyst forum held on December 9, 2019. This
study demonstrated that Libervant provided similar systemic
diazepam exposures to the reference drug and exhibited
significantly less variability. The study supports the use of
Libervant as an easily-administered treatment for patients with
epilepsy experiencing seizure emergencies. A replay of this
forum is available at
https://investors.aquestive.com/events-and-presentations.
In a recent decision, the FDA’s Center for Drug
Evaluation and Research granted marketing exclusivity for seven
years to another drug approved for the labeled indication of 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 six years of age and older. Although we cannot
be assured of the FDA’s approval of Libervant or finding that
Libervant represents a “major contribution to patient care” to
overcome this market exclusivity, Aquestive remains committed to
helping people affected by seizure clusters by looking to bring
important innovative products to the market that will improve the
lives of patients. See below for a further explanation of the
FDA’s criteria for determining “major contribution to patient care”
in “Additional Information Regarding Orphan Drug Exclusivity.”
About Aquestive
TherapeuticsAquestive Therapeutics is a specialty
pharmaceutical company that applies innovative technology to solve
therapeutic problems and improve medicines for patients. Aquestive
is advancing a late-stage proprietary product pipeline to treat CNS
conditions and 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 StatementThis
press release includes 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 may
include, but are not limited to, statements regarding therapeutic
benefits and plans and objectives for regulatory approvals of
Libervant and our other product candidates; ability to obtain FDA
approval and advance Libervant and our other product candidates to
the market; statements about our growth and future financial and
operating results and financial position, regulatory approval and
pathways, clinical trial timing and plans, our and our competitors’
orphan drug approval and resulting drug exclusivity for our
products or products of our competitors, short-term and long-term
liquidity and cash requirements, cash funding and cash burn,
business strategies, market opportunities, and other statements
that are not historical facts.
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 and plans; risk of
delays in FDA approval of Libervant and our other drug candidates
or failure to receive approval; risk that a competitor obtains FDA
orphan drug exclusivity for a product with the same active moiety
as the orphan drug product for which we are seeking FDA approval
and that such earlier approved competitor orphan drug blocks our
product in the U.S. for seven years for the same indication; risk
of our ability to demonstrate to the FDA “clinical superiority”
within the meaning of FDA regulations of our drug candidate
Libervant relative to the FDA-approved Valtoco® (diazepam nasal
spray) and Diastat® (diazepam rectal gel) including by establishing
a major contribution to patient care within the meaning of FDA
regulations relative to the approved product and there can be no
assurance that we will be successful; 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 cash requirements and other cash
needs, at the times and in the amounts needed; risk of failure to
satisfy all financial and other debt covenants and of any default;
risk related to government claims against Indivior for which we
license, manufacture and sell Suboxone and which accounts for the
substantial part of our current operating revenues; risks
associated with Indivior’s announcement of its intention to cease
production of its authorized generic buprenorphine naloxone film
product, including the impact from loss of orders for the
authorized generic product and risk of eroding market share for
Suboxone and risk of sunsetting product; risks related to the
outsourcing of certain sales, marketing and other operational and
staff functions to third parties; risk of the rate and degree of
market acceptance of our products and product candidates; the
success of any competing products, including generics; risk of the
size and growth of our product markets; risk 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 action 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 governmental 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 risks and
uncertainties affecting the Company including those described in
the “Risk Factors” section and in other sections included in the
Company's Annual Report on Form 10‑K filed with the SEC on March
14, 2019, in our quarterly reports on Form 10-Q, and in the Form
8-K filed on January 13, 2020. Given these 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.
Additional Information Regarding Orphan
Drug ExclusivityThe FDA’s response to the Company’s
Citizen’s Petition dated November 1, 2019 includes the following in
discussing orphan drug exclusivity, including pertinent factors
that may be considered by the FDA in making a determination of
“major contribution to patient care” for “clinical superiority” as:
convenient treatment location; duration of treatment; patient
comfort; reduced treatment burden; advances in ease and comfort of
drug administration; longer periods between doses; and potential
for self-administration:
Section 527 of the [Federal Food, Drug, and
Cosmetic Act] defines “clinically superior” to mean “the drug
provides a significant therapeutic advantage over and above an
already approved or licensed drug in terms of greater efficacy,
greater safety, or by providing a major contribution to patient
care.” The orphan-drug regulations elaborate on the definition of
“clinically superior” as follows:
Clinically superior means that a drug is shown
to provide a significant therapeutic advantage over and above that
provided by an approved drug (that is otherwise the same drug) in
one or more of the following ways: Greater effectiveness than an
approved drug (as assessed by effect on a clinically meaningful
endpoint in adequate and well controlled clinical trials).
Generally, this would represent the same kind of evidence needed to
support a comparative effectiveness claim for two different drugs;
in most cases, direct comparative clinical trials would be
necessary; or Greater safety in a substantial portion of the target
populations, for example, by the elimination of an ingredient or
contaminant that is associated with relatively frequent adverse
effects. In some cases, direct comparative clinical trials will be
necessary; or In unusual cases, where neither greater safety
nor greater effectiveness has been shown, a demonstration that the
drug otherwise makes a major contribution to patient
care.
Because of the diverse ways in which drugs may
qualify as clinically superior under these criteria, FDA evaluates
clinical superiority on a case by case basis. Specifically, with
respect to the major contribution to patient care prong of the
clinical superiority definition, the FDA has further stated:
There is no way to quantify such superiority in a general way. The
amount and kind of superiority needed would vary depending on many
factors, including the nature and severity of the disease or
condition, the quality of the evidence presented, and diverse other
factors; and The following factors, when applicable to severe or
life-threatening diseases, may in appropriate cases be taken into
consideration when determining whether a drug makes a major
contribution to patient care: convenient treatment location;
duration of treatment; patient comfort; reduced treatment burden;
advances in ease and comfort of drug administration; longer periods
between doses; and potential for self-administration.
__________________________________
PharmFilm® and the Aquestive logo are registered
trademarks of Aquestive Therapeutics, Inc. All other
registered trademarks referenced herein are the property of their
respective owners.
Media Inquiries: Mark
Corbaemark.corbae@icrinc.com858.213.7956
Investor Inquiries: Stephanie Carrington
stephanie.carrington@icrinc.com 646-277-1282
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