Analyst Sees Path Forward After FDA Rejects Aquestive Drug

Aquestive Therapeutics, a New Jersey-based drug developer, received a rejection notice from the U.S. Food and Drug Administration on January 30, 2026, for its Anaphylm product, an under-the-tongue epinephrine film designed to treat severe allergic reactions. Leerink Partners, a health care investment firm, described the decision as having a silver lining because the FDA outlined specific steps for the company to fix the problems and resubmit.

Background

Aquestive focuses on products that deliver drugs through the mouth rather than needles or pills. Anaphylm aims to offer a needle-free option for people facing anaphylaxis, a life-threatening allergy response that needs quick epinephrine. Current treatments rely on injections from devices like EpiPens, which some patients find hard to carry or use in a panic.

The company filed its application for Anaphylm approval last year. The FDA set a review deadline of January 31, 2026. On the day before, agency staff sent a Complete Response Letter, or CRL, which blocks approval until issues are addressed. This letter pointed to problems only in how the product is packaged and how people would use it at home. No concerns came up about the drug's safety in repeated doses, how it works in the body, or manufacturing quality.

Aquestive ran 11 studies with nearly 1,000 doses given to test Anaphylm. Market tests showed 96 percent of users preferred it over injections when they could open and handle it right. The firm held a call with investors on February 2, 2026, to explain the situation. Shares rose that day, signaling some market confidence.

Key Details

The CRL listed fixes needed in human factors, which cover how real people interact with the product. This includes the pouch that holds the film, directions for opening it, and labels. The FDA also wants one more study on drug levels in the blood, called pharmacokinetics or PK, to back up the main data.

Company Response Plan

Aquestive plans changes to the pouch design, clearer instructions, and updated labels. It will run a new human factors study to prove users can handle these updates without mistakes. The PK study will provide extra support. Leaders expect to finish both quickly and file again in the third quarter of 2026. They asked for a Type A meeting with the FDA soon to map the fastest route. The company wants priority review, which could cut FDA time to six months.

Daniel Barber, president and chief executive of Aquestive, spoke on the investor call.

"While it is unfortunate to have received a CRL, we believe that, with the clarity we now have from the FDA, we have made significant progress toward approval. We are encouraged that the issues in the letter are limited to human factors and a supportive PK study, once human factors are addressed."

Gary Slatko, interim chief medical officer, called the letter a major step to reduce risks. He noted it clears doubts on key safety and performance points. Sherry Korczynski, chief commercial officer, said the company stays ready for launch but will hold off hiring a sales team until approval. The epinephrine market still runs on injectors, she added.

Leerink analysts highlighted the limited scope of issues as a positive sign. They pointed out no manufacturing or clinical trial demands, which often delay drugs longer. This clear path lets Aquestive focus work and avoid big setbacks.

Aquestive ended last quarter with enough cash to cover resubmission, approval push, and early sales setup. It expects strong funds through 2026.

What This Means

For Aquestive, the CRL delays U.S. sales of Anaphylm but sets a roadmap without demanding new big trials. Resubmission in Q3 2026 could lead to approval by late 2026 or early 2027 if the FDA moves fast. Success here would give patients an easy-to-store, non-injectable rescue option. The firm sees demand from those who skip carrying EpiPens due to bulk or fear of needles.

Globally, Aquestive pushes ahead. It started talks with regulators in Canada, Europe, and the U.K. in 2025. Europe's agency said no extra trials needed before filing. Canada and Europe filings come in the second half of 2026. U.K. feedback arrives soon. These steps could open markets even if U.S. approval lags.

Investors watch closely. The stock bump shows belief in the plan. Leerink's view of a silver lining rests on the narrow fixes and cash buffer. Risks remain if the FDA wants more changes or slows review. Human factors studies sometimes uncover wider use issues, though Aquestive's experience suggests smooth fixes.

Patients with allergies gain from options like Anaphylm. Needle-free forms could boost quick response in emergencies. Aquestive's tech applies to other drugs too, so this outcome shapes its future pipeline.

The company keeps focus on Anaphylm while advancing other mouth-dissolving products. Barber stressed patient needs drive their work. With FDA guidance now in hand, Aquestive moves to meet it step by step.