Aquestive Therapeutics, Inc. (NASDAQ: AQST), a specialty
pharmaceutical company focused on developing and commercializing
differentiated products that meet patients’ unmet needs and solve
therapeutic problems, today reported that the U.S. Food and Drug
Administration (FDA) issued a response letter (Response) dated
January 10, 2020 denying Aquestive’s Citizen’s Petition received by
the FDA on November 1, 2019, including the supplement to the
Citizen’s Petition received by the FDA on December 4, 2019 (Docket
No. FDA-2019-P-5121) (Petition). The Petition requested,
among other things, that the FDA stay approval of a New Drug
Application for Valtoco® (diazepam nasal spray) submitted by
Neurelis, Inc. until additional clinical studies were
conducted. In the Response, the FDA indicated that it had
approved Neurelis’s NDA for Valtoco on January 10, 2020.
Valtoco has received orphan drug exclusivity from the FDA’s Center
for Drug Evaluation and Research commencing as of January 10, 2020
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.
“This patient population has been underserved
for some time with little choice beyond the rectally administered
gel and choice is important. We believe that our candidate
drug Libervant™ (diazepam) Buccal Film will, if approved by the
FDA, further expand patient choice as the first orally administered
dosage form for this patient population,” said Keith J. Kendall,
Chief Executive Officer of Aquestive.
“We appreciate that the FDA has confirmed in the
Response its guidance that 505(b)(2) drugs can be approved without
proving bioequivalence if they demonstrate “relative
bioavailability” to the reference drug. The FDA stated in the
Response, when granting exclusivity based upon “major contribution
to patient care” over and above already approved products for the
indication, 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. In making this determination for
Valtoco, the FDA indicated in the Response that the intranasal
route of administration provides a major contribution to patient
care over the rectal route of administration by providing a
significantly improved ease of use.”
“We look forward to working with the FDA in the
coming months 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,” concluded Mr. Kendall.
Although we cannot be assured of FDA approval of
Libervant, 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.
We provided preliminary 2020 revenue guidance in
our press release dated January 10, 2020. As a reminder, we
did not include any Libervant revenues in that guidance.
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 Statement This
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® (diazepam) Buccal Film 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 Exclusivity
The Response includes the following in
discussing orphan drug exclusivity:
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; orGreater 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;andThe 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. |
The Aquestive logo is a registered trademarks of
Aquestive Therapeutics, Inc. All other registered trademarks
referenced herein are the property of their respective owners.
Media Inquiries: Mark Corbaee: mark.corbae@icrinc.comp:
858.213.7956
Investor Inquiries: Stephanie Carrington
stephanie.carrington@icrinc.com 646-277-1282
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