Aquestive Therapeutics Receives Complete Response Letter from FDA for Libervant™ (diazepam) Buccal Film for Management of S...
September 25 2020 - 5:38PM
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 the U.S. Food and Drug
Administration (FDA) has issued a complete response letter (CRL)
regarding the New Drug Application (NDA) for Libervant™ (diazepam)
Buccal Film for management of seizure clusters. The FDA issues a
CRL to indicate that the review cycle for an application is
complete but the application cannot be approved in its current
form.
In the CRL, the FDA cited that, in a study
submitted by the Company with the NDA, certain weight groups showed
a lower drug exposure level than desired. The Company intends to
provide to the FDA additional information on PK modeling to
demonstrate that dose adjustments will obtain the desired exposure
levels. There were no other safety, clinical
pharmacology/biopharmaceutics or CMC issues identified in the CRL.
The FDA did cite a small number of protocol deviations in blood
draws in one of the studies in the NDA. The Company believes, based
on discussions with the FDA, that the Company will not need to
conduct any further clinical studies in order to cure the items
cited in the CRL, and will confirm that view in its upcoming
follow-up meeting with the FDA.
Based on interactions with the Agency, the
Company believes that this CRL will not be a barrier to ultimate
approval, as the CRL was limited to providing additional
information on PK modeling for an adjusted dosing regimen for a
limited subset of patient weight categories. The Company plans to
request a Type A meeting with the FDA in the coming weeks and to
resubmit the NDA prior to the end of 2020 with the adjusted dosage
regimen for the identified weight groups at issue. A submission
before the end of the year should result in a PDUFA Action Date in
the 1st half of 2021. The Agency did not include any indication
regarding approval of U.S. market access for Libervant at this
time.
“While we are surprised by and disappointed with
the Agency’s decision, we remain committed to continuing to work
with the FDA toward approval of Libervant to provide epilepsy
patients with the first orally administered treatment for
breakthrough and seizure clusters,” said Keith J. Kendall,
President and Chief Executive Officer of Aquestive. “We look
forward to quickly scheduling a meeting with the FDA to solidify
Libervant’s path forward and in-turn move toward the NDA
resubmission before year’s end. Epilepsy patients have been
underserved for some time with little choice beyond device-based
products such as rectally administered gels and nasal sprays and
Libervant represents a meaningful and improved therapy for patients
who can’t or won’t use the alternatives. We believe that the
Company will be able to provide the necessary data to the FDA to
allow for Libervant’s approval,” concluded Mr. Kendall.
Conference Call on September
25, 2020 at 6:30
p.m. ETThe Company will host a
conference call today, September 25, 2020 at 6:30 p.m. to discuss
the FDA’s decision. To access the conference call dial (866)
417-5886 from the U.S. and (409) 217-8235 internationally, followed
by the conference ID: 3205078. The Company has prepared FAQs and
other materials for discussion during the conference call which
have been filed today under a Form 8-K filed with the United States
Securities and Exchange Commission. A live webcast and these
materials will be available on the Investors section of the
Company’s website at
https://investors.aquestive.com/events-and-presentations. The
webcast and these materials will be archived for 30 days.
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. The Company
believes 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.
About Aquestive
TherapeuticsAquestive Therapeutics is a 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
include, but are not limited to, statements regarding therapeutic
benefits and plans and objectives for regulatory approvals of
Libervant™, ability to cure the deficiencies identified in the
FDA’s Complete Response Letter dated September 25, 2020 regarding
the New Drug Application for Libervant and obtain FDA approval of
Libervant for U.S. market access, timing of FDA review and approval
of Libervant, pathways, clinical trials, and plans for approval of
Libervant, our and our competitors’ orphan drug approval and
resulting drug exclusivity for Libervant or products of our
competitors. These forward-looking statements are also 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 ability to obtain FDA approval and advance
Libervant, AQST-108 and our other product candidates to the market,
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 of our ability to demonstrate to the FDA “clinical
superiority” within the meaning of the FDA regulations of our drug
candidate 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 inherent in commercializing a new product
(including technology risks, financial risks, market risks and
implementation risks and regulatory limitations); risks and
uncertainties concerning any potential monetization of royalty and
other revenue stream of KYNMOBI (apomorphine) and of sufficiency of
net proceeds of any such monetization after satisfaction of and
compliance with 12.5% Senior Notes obligations, as applicable; 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; risk associated
with Indivior’s cessation of 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 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 (SEC). 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® and the
Aquestive logo are registered trademarks of Aquestive Therapeutics,
Inc. All other registered trademarks referenced herein are the
property of their respective owners.
Investor Inquiries:Stephanie
Carringtonstephanie.carrington@icrinc.com646-277-1282
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