Aquestive Therapeutics, Inc. (NASDAQ: AQST) has announced key milestones for its pipeline products, including Anaphylm™ (epinephrine) Sublingual Film and AQST-108 (epinephrine) Topical Gel, alongside updates on Libervant® (diazepam) Buccal Film. The company is focused on innovative delivery technologies to improve patient outcomes.
Anaphylm Sublingual Film NDA Submission on Track
Aquestive is preparing to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Anaphylm in the first quarter of 2025. Anaphylm is designed as a sublingual film for the treatment of severe allergic reactions, including anaphylaxis. If approved, Anaphylm would be the first and only orally delivered epinephrine product, offering a needle-free alternative to current standard-of-care treatments.
The company has completed a successful pre-NDA meeting with the FDA, gaining clear guidance on the regulatory pathway. Aquestive has also initiated a Phase 1 pediatric trial of Anaphylm in children aged 7 to 17 years and ≥30 kg, demonstrating its commitment to expanding access to this treatment across age groups.
AQST-108 Phase 2a Trial for Alopecia Areata
Aquestive is advancing its AQST-108 topical gel for the treatment of alopecia areata (AA), a dermatological condition affecting an estimated 6.7 million people in the United States. The company successfully completed a pre-Investigational New Drug (IND) meeting with the FDA in December 2024, receiving supportive feedback for continued development. Aquestive remains on track to begin its Phase 2a trial in patients with alopecia areata in the second quarter of 2025.
Existing therapies for alopecia areata, such as Janus kinase (JAK) inhibitors, are systemic treatments with potential side effects, including "black box" warnings. AQST-108, a topically delivered adrenergic agonist prodrug gel, demonstrated no serious adverse events or topical adverse events in a first-in-human Phase 1 clinical trial. Because AQST-108 is topical and acts at the application site, it may avoid the systemic side effects associated with current treatments. The market opportunity for JAK inhibitors in alopecia areata is estimated to be over one billion U.S. dollars, suggesting a significant potential market for AQST-108 if approved.
Libervant Market Expansion
Libervant, a buccal film formulation of diazepam, received FDA approval in 2024 for treating seizure clusters in pediatric patients with epilepsy between two and five years of age. Aquestive received Orphan Drug Exclusivity for Libervant for patients between two and five years of age until April 2031. Libervant is the first and only FDA-approved orally administered rescue product for this patient population.
Dan Barber, President and Chief Executive Officer of Aquestive, stated, "We believe our long-term growth strategy remains compelling with the potential approval and launches of Anaphylm, Libervant (patients 6+), and AQST-108 in the U.S. and around the world."
