Aquestive Therapeutics Announces Positive FDA Feedback and Reaffirms NDA Submission Guidance for Anaphylm™ (epinephrine) Sublingual Film
Aquestive Therapeutics (NASDAQ: AQST) received positive FDA feedback regarding its planned NDA submission for Anaphylm™, a potential first-ever orally delivered epinephrine product for severe allergic reactions treatment. The FDA agreed with the company's planned NDA content, format, safety evaluation, and pediatric trial design, which has begun in the U.S. and Canada. The company reaffirmed its NDA submission timeline for Q1 2025, with no additional adult clinical trials required. The FDA indicated a possible advisory committee meeting due to the novel route of administration. Anaphylm aims to serve an estimated 33 million Americans with food allergies who currently rely on epinephrine medical devices.
Aquestive Therapeutics (NASDAQ: AQST) ha ricevuto un feedback positivo dalla FDA riguardo alla sua prevista submission NDA per Anaphylm™, un potenziale prodotto di epinefrina da somministrare per via orale per il trattamento delle reazioni allergiche gravi. La FDA ha concordato con il contenuto, il formato, la valutazione di sicurezza e il design della sperimentazione pediatrica previsti dall'azienda, che è già iniziata negli Stati Uniti e in Canada. L'azienda ha confermato la sua tempistica per la submission NDA nel primo trimestre del 2025, senza la necessità di ulteriori trial clinici sugli adulti. La FDA ha indicato una possibile riunione del comitato consultivo a causa della nuova via di somministrazione. Anaphylm mira a servire circa 33 milioni di americani con allergie alimentari che attualmente si affidano a dispositivi medici per epinefrina.
Aquestive Therapeutics (NASDAQ: AQST) recibió comentarios positivos de la FDA sobre su presentación NDA planificada para Anaphylm™, un posible producto de epinefrina administrado por vía oral para el tratamiento de reacciones alérgicas severas. La FDA estuvo de acuerdo con el contenido, formato, evaluación de seguridad y diseño de ensayo pediátrico propuestos por la compañía, que han comenzado en EE. UU. y Canadá. La empresa reafirmó su cronograma de presentación de NDA para el primer trimestre de 2025, sin necesidad de ensayos clínicos adicionales en adultos. La FDA indicó una posible reunión del comité asesor debido a la nueva vía de administración. Anaphylm tiene como objetivo atender a aproximadamente 33 millones de estadounidenses con alergias alimentarias que actualmente dependen de dispositivos médicos de epinefrina.
Aquestive Therapeutics (NASDAQ: AQST)는 중증 알레르기 반응 치료를 위한 잠재적인 최초의 경구용 에피네프린 제품인 Anaphylm™에 대한 NDA 제출 계획에 대해 FDA로부터 긍정적인 피드백을 받았습니다. FDA는 회사가 계획한 NDA 내용, 형식, 안전성 평가 및 소아 시험 설계에 동의했으며, 이는 미국과 캐나다에서 이미 시작되었습니다. 이 회사는 추가적인 성인 임상 시험 없이 2025년 1분기로 NDA 제출 일정이 확정되었다고 밝혔습니다. FDA는 새로운 투여 경로로 인해 자문위원회 회의의 가능성을 언급했습니다. Anaphylm은 현재 에피네프린 의료 장치에 의존하는 약 3,300만 명의 미국 식품 알레르기를 가진 사람들을 돕는 것을 목표로 하고 있습니다.
Aquestive Therapeutics (NASDAQ: AQST) a reçu des retours positifs de la FDA concernant sa soumission NDA prévue pour Anaphylm™, un potentiel premier produit d'épinéphrine administré par voie orale pour le traitement des réactions allergiques sévères. La FDA a approuvé le contenu, le format, l'évaluation de la sécurité et la conception de l'essai pédiatrique proposés par l'entreprise, qui ont déjà commencé aux États-Unis et au Canada. L'entreprise a réaffirmé son calendrier de soumission NDA pour le premier trimestre 2025, sans nécessiter d'essais cliniques supplémentaires chez les adultes. La FDA a indiqué une possible réunion du comité consultatif en raison de la nouvelle voie d'administration. Anaphylm vise à servir environ 33 millions d'Américains souffrant d'allergies alimentaires qui dépendent actuellement des dispositifs médicaux épinéphrine.
Aquestive Therapeutics (NASDAQ: AQST) erhielt positives Feedback von der FDA hinsichtlich der geplanten NDA-Einreichung für Anaphylm™, einem potenziell ersten oral verabreichten Epinephrin-Produkt zur Behandlung schwerer allergischer Reaktionen. Die FDA stimmte dem geplanten Inhalt, Format, der Sicherheitsbewertung und dem Design der pädiatrischen Studie des Unternehmens zu, die in den USA und Kanada begonnen hat. Das Unternehmen bekräftigte seinen Zeitplan zur NDA-Einreichung für das erste Quartal 2025, ohne dass zusätzliche klinische Studien an Erwachsenen erforderlich sind. Die FDA deutete auf ein mögliches Treffen des Beratungsausschusses hin, bedingt durch die neuartige Verabreichungsform. Anaphylm soll etwa 33 Millionen Amerikanern mit Nahrungsmittelallergien dienen, die derzeit auf Epinephrin-Medizinprodukte angewiesen sind.
- FDA confirms no additional adult clinical trials needed before NDA submission
- FDA agrees with planned NDA content, format, and safety evaluation
- Pediatric trial successfully commenced in U.S. and Canada
- Company maintains Q1 2025 NDA submission timeline
- Product targets large market of 33 million Americans with food allergies
- FDA may require advisory committee meeting due to new route of administration
- FDA requested modifications to pediatric trial protocol
Insights
This FDA feedback represents a significant milestone for Aquestive's Anaphylm program. The confirmation that no additional adult clinical trials are needed and alignment on NDA submission content substantially de-risks the regulatory pathway. The potential first-in-class oral epinephrine delivery system addresses a critical market gap, targeting 33 million Americans with food allergies who currently rely solely on injectable devices.
The FDA's focus on pharmacokinetic sustainability and potential advisory committee meeting suggests thorough scrutiny of this novel route of administration. The commenced pediatric trial and clear regulatory guidance strengthen the path to Q1 2025 NDA submission. The market opportunity is substantial, as current epinephrine auto-injectors face persistent challenges with accessibility, compliance and ease of use.
The positive FDA feedback significantly strengthens Aquestive's market position in the $2+ billion epinephrine market. The sublingual film format could revolutionize anaphylaxis treatment by addressing key barriers to current auto-injectors: portability, ease of use and patient compliance. This competitive advantage could drive substantial market share capture if approved.
The clear regulatory pathway reduces investment risk and positions Aquestive to potentially disrupt the dominated by EpiPen and its alternatives. The pediatric trial initiation also demonstrates progress toward accessing the important childhood allergy market segment. The stock should benefit from reduced regulatory uncertainty and enhanced commercialization potential.
- Reaffirms New Drug Application (NDA) first quarter 2025 submission guidance
- Confirms no additional adult clinical trials are necessary prior to NDA submission
- Commenced pediatric trial in the U.S. and Canada
WARREN, N.J., Dec. 02, 2024 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies (“Aquestive,” the “Company” or “we”), today announced receipt of positive feedback from the U.S. Food and Drug Administration (FDA) prior to its planned NDA submission for Anaphylm™ (epinephrine) Sublingual Film. Anaphylm has the potential to be the first and only orally delivered epinephrine product for the treatment of severe allergic reactions, including anaphylaxis, if approved by the FDA.
“We believe FDA alignment on the completeness of our adult development program for Anaphylm is a major milestone for the Company and the allergy community,” said Daniel Barber, President and Chief Executive Officer of Aquestive. “We have commenced our single-dose pediatric trial in December 2024, and we believe we have a clear path to an NDA submission in the first quarter of 2025. Currently, the estimated 33 million Americans with food allergies only have the option of carrying epinephrine medical devices. Our engagement with the allergy community continues to inform us that bringing the first and only orally delivered epinephrine product for anaphylaxis to market can potentially be transformative for patients. This patient community deserves continued innovation that creates easy-to-carry, easy-to-administer alternatives to today’s approved medical devices.”
In a written response, the FDA agreed with the Company’s planned NDA content and format for the submission, planned safety evaluation, and planned pediatric trial, which has commenced at both U.S. and Canadian sites. The FDA also provided further guidance on additional data views to be included in the planned NDA submission and continued to emphasize their focus on pharmacokinetic sustainability for a single dose. In addition, the FDA requested minor modifications to the Company’s pediatric trial protocol. We have incorporated these FDA requested changes in the final pediatric trial protocol and do not expect these changes to have any significant impact on the planned timing of the pediatric trial. Finally, the FDA noted that due to the new route of administration and the data supporting this route of administration, an advisory committee meeting may be necessary. Further details regarding the FDA’s comments are available on the Events and Presentation section of the Investor page on the Aquestive website.
About Anaphylm™ (epinephrine) Sublingual Film
Anaphylm™ (epinephrine) Sublingual Film is a polymer matrix-based epinephrine prodrug product candidate. Anaphylm 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 Anaphylm 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 Anaphylm trade name for AQST-109 has been conditionally approved by the FDA. Final approval of the Anaphylm proprietary name is conditioned on FDA approval of the product candidate.
About Aquestive Therapeutics
Aquestive is a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies. We are developing orally administered products to deliver complex molecules, providing novel alternatives to invasive and inconvenient standard of care therapies. Aquestive has five commercialized products marketed by the Company and its licensees in the U.S. and around the world, and is the exclusive manufacturer of these licensed products. The Company also collaborates with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven drug development and commercialization capabilities. Aquestive is advancing a late-stage proprietary product candidate for the treatment of severe allergic reactions, including anaphylaxis, and an earlier stage epinephrine prodrug topical gel for various possible dermatology conditions. For more information, visit Aquestive.com and follow us on LinkedIn.
Forward-Looking Statement
Certain 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,” “may,” 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 our product candidate Anaphylm™ (epinephrine) Sublingual Film through clinical development and approval by the FDA, including the timing of submission of a pediatric clinical trial and NDA for Anaphylm with the FDA, and the anticipated launch of Anaphylm, if approved by the FDA; that the results of the Company’s clinical studies for Anaphylm are sufficient to support submission of the NDA for approval of Anaphylm by the FDA; the potential indications and potential benefits our products and product candidates could bring to patients; and 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 our development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans, including those relating to Anaphylm (including for pediatric patients); risk of delays in advancement of the regulatory approval process through the FDA of our product candidates, including the filing of the respective NDAs, including for Anaphylm, or the failure to receive FDA approval at all of any of these product candidates; risk of the Company’s ability to generate sufficient clinical data for approval of our product candidates, including with respect to our pharmacokinetic and pharmacodynamic comparability submission for FDA approval of Anaphylm; risk of the Company’s ability to address the FDA’s comments on the Company’s clinical trials and other guidance including those identified in the FDA Type C meeting minutes and pre-NDA minutes for Anaphylm, including the risk that the FDA may require additional clinical studies for approval of Anaphylm; risk of the success of any competing products; risks and uncertainties inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risk of sufficient capital and cash resources, including sufficient access to available debt and equity financing, including under our ATM facility and the Lincoln Park Purchase Agreement, 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, including to fund commercialization activities relating to fund future clinical development and commercial activities for our product candidates, including Anaphylm, should these product candidates be approved by the FDA; risk of eroding market share for Suboxone® and risk as a sunsetting product, which accounts for the substantial part of our current operating revenue; risk of default of our debt instruments; 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 in the U.S. of Anaphylm and our other product candidates, should these product candidates be approved by the FDA, and for our licensed products in the U.S. and abroad; risk of 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 our products; risk that our patent applications for our product candidates, including for Anaphylm, will not be timely issued, or issued at all, by the U.S. Patent and Trademark Office; risk of unexpected patent developments; risk of legislation and regulatory actions and changes in laws or regulations affecting our business including relating to our products and products candidates and product pricing, reimbursement or access therefor; risk of loss of significant customers; risks related to claims and legal proceedings against Aquestive including patent infringement, securities, business torts, investigative, product safety or efficacy and antitrust litigation matters; risk of product recalls and withdrawals; risks related to any disruptions in our information technology networks and systems, including the impact of cybersecurity attacks; risk of increased cybersecurity attacks and data accessibility disruptions due to remote working arrangements; risk of adverse developments affecting the financial services industry; risks related to inflation and rising interest rates; risks related to the impact of any global pandemic and other pandemic diseases on our business, including with respect to our clinical trials and the site initiation, patient enrollment and timing and adequacy of those clinical trials, regulatory submissions and regulatory reviews and approvals of our product candidates, availability of pharmaceutical ingredients and other raw materials used in our products and product candidates, supply chain, manufacture and distribution of our products and product candidates; risks and uncertainties related to general economic, political (including the Ukraine and Israel wars and other acts of war and terrorism), business, industry, regulatory, financial and market conditions and other unusual items; and other uncertainties affecting us including those described in the "Risk Factors" section and in other sections included in the Company’s Annual Report on Form 10-K as of and for the year ended December 31, 2023, subsequent Quarterly Reports on Form 10-Q, and subsequent Current Reports on Form 8-K filed with the U.S. Securities and 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 the Company or any person acting on its 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.
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FAQ
When will Aquestive Therapeutics (AQST) submit the NDA for Anaphylm?
What is unique about Aquestive's Anaphylm compared to existing treatments?
Does AQST need additional adult clinical trials for Anaphylm's NDA submission?
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