Aquestive Therapeutics Inc., (NASDAQ: AQST) revealed that the US FDA issued a Complete Response Letter (CRL) for its New Drug Application (NDA) for Anaphylm (dibutepinephrine) sublingual film. The innovative epinephrine treatment, designed for patients weighing 30kg or more experiencing Type I allergic reactions, encountered regulatory challenges focused on human factors and packaging design.
The FDA’s Complete Response Letter primarily centered on human factors validation study deficiencies, identifying potential safety risks in emergency anaphylaxis treatment scenarios. Specific concerns included: difficulties in pouch opening, incorrect film placement, and potential patient usability challenges
These issues could compromise the critical rapid-response nature of anaphylaxis treatment, prompting the FDA to request modifications to packaging design, instructions for use, and pouch and carton labeling.
Anaphylm represents a novel approach to emergency allergy treatment, offering a postage stamp-sized sublingual film that dissolves rapidly without water, weighs less than an ounce, can be easily carried in a pocket, and provides non-invasive epinephrine delivery. The product’s polymer matrix-based prodrug technology aims to address significant limitations in current epinephrine auto-injector designs, potentially improving patient accessibility and treatment compliance.
Following the FDA CRL, Aquestive plans an aggressive regulatory engagement strategy including requesting a Type A meeting with the FDA, conducting a modified human factors validation study, and completing supplemental pharmacokinetic research. The firm is targeting a resubmission by Q3 2026.