TLDR
- FDA declined approval for Aquestive Therapeutics’ Anaphylm oral film treatment for severe allergic reactions on January 30, 2026
- Rejection focused on packaging and usability issues, not drug efficacy, after users struggled to open pouches and place film correctly in validation study
- Aquestive has modified pouch opening, instructions, and labeling and plans new human factors validation study
- FDA requested additional blood-level study to check if packaging changes affect drug absorption
- Company expects to resubmit application as early as Q3 2026 and plans regulatory filings in Europe and Canada in H2 2026
Aquestive Therapeutics received a Complete Response Letter from the FDA on January 30, rejecting its application for Anaphylm sublingual film. The oral treatment was designed for severe allergic reactions including anaphylaxis in patients weighing 30kg or more.
Aquestive Therapeutics, Inc., AQST
The rejection represents a setback for the company’s efforts to provide an alternative to existing treatments. Current options include ARS Pharma’s nasal spray Neffy and injectable epinephrine auto-injectors like EpiPen.
The FDA’s concerns centered on how patients would use the product during emergencies. A human factors validation study revealed problems with the packaging design. Some participants struggled to open the pouch quickly enough.
Others placed the film incorrectly in their mouths. The regulator determined these issues could create safety risks when patients experience anaphylaxis. Anaphylaxis is a rapidly occurring, life-threatening allergic response triggered by foods, insect stings, or medications.
The FDA did not question the drug’s effectiveness. The Complete Response Letter focused exclusively on administration and labeling issues. Manufacturing and chemistry data submitted with the application also passed without concerns.
Company Response and Timeline
Aquestive has already implemented changes to address the FDA’s feedback. The company modified the pouch opening mechanism to make it easier to access. It updated the instructions for use and revised both pouch and carton labels.
The company plans to conduct a new human factors validation study with these modifications. This study will test whether the changes resolve the usability problems identified by the FDA.
The regulator also requested one additional blood-level study. This pharmacokinetic study will determine if the packaging and labeling changes affect how the body absorbs the drug.
Market Reaction and Future Plans
Aquestive shares jumped over 17% in premarket trading following the announcement. Investors apparently viewed the rejection as addressable rather than a fundamental problem with the drug.
The company believes it can resolve all deficiencies identified in the letter. Aquestive expects to resubmit the application as early as the third quarter of 2026.
The FDA had previously flagged labeling deficiencies in January 2026. The Complete Response Letter provided more detailed feedback on these issues.
Aquestive also plans international expansion for Anaphylm. The company will pursue regulatory filings in Europe and Canada during the second half of 2026. It expects feedback from the UK regulator in the first quarter of 2026.
The company confirmed that comparability data submitted with the original application remains valid. This includes bracketing studies, repeat dose testing, and sustainability data.
Get 3 Free Stock Ebooks
Discover top-performing stocks in AI, Crypto, and Technology with expert analysis.
- Top 10 AI Stocks - Leading AI companies
- Top 10 Crypto Stocks - Blockchain leaders
- Top 10 Tech Stocks - Tech giants
