Aquestive Therapeutics (AQST) Status update summary

FDA feedback and regulatory update

• FDA issued a Complete Response Letter (CRL) for Anaphylm, citing deficiencies limited to packaging, administration, and human factors, but no issues with pharmacokinetics, safety, efficacy, or CMC.

• Human factors concerns included difficulty opening the pouch, incorrect film placement, potential tearing, and premature removal due to taste or sensation; most errors were linked to lack of instructions.

• The company will revise packaging, labeling, and Instructions for Use for clarity and usability, incorporating FDA feedback and additional risk controls.

• No safety or efficacy concerns were raised, and all adverse events in trials were mild and self-resolving.

• A pending citizen petition is under FDA review but does not affect current approvability comments.

Required actions and clinical plans

• A new human factors validation study (about 75 participants) and a PK study (18-24 per arm) are required, addressing alternate administration scenarios and FDA requests.

• PK studies must evaluate the impact of use errors such as chewing, incorrect placement, and water intake prior to administration.

• The company has designed protocols for these studies, to be finalized after a Type A meeting with the FDA in Q1–Q2 2026.

• NDA resubmission is targeted for Q3 2026, classified as Class 2, with a request for expedited review.

• No additional clinical trials or CMC issues were raised by the FDA.

Commercial and market outlook

• Commercial launch preparations and salesforce recruitment will align with the new approval timeline, post-approval.

• Market research shows strong patient preference for Anaphylm, with 96% choosing it in demos.

• The market for epinephrine products is growing, and the company expects to be commercial-ready.

• Educational materials, demo units, and training will be provided to healthcare providers and patients.

• The company remains well-capitalized and expects to end 2026 with significant cash, with sufficient funding for approval and pre-launch processes.