FDA Rejects Initial Needle-Free EpiPen Alternative, Calls for Further Research


The Food and Drug Administration (FDA) has rejected approval for an epinephrine nasal spray called Neffy, which would have provided a needle-free alternative to epinephrine autoinjectors, such as EpiPens. The FDA’s decision comes despite its advisory committee having recommended approval of the drug for use in both children and adults in May, a rare circumstance in which the FDA does not typically approve drugs endorsed by its committees.

The rejection is based on the FDA’s request for additional data to support Neffy’s approval, specifically a study comparing Neffy to an epinephrine autoinjector in individuals with allergen-induced nasal symptoms like sneezing, itching, and congestion. The agency’s primary concern has been the lack of clinical data on Neffy’s effectiveness, especially its untested use in treating anaphylaxis, a severe allergic reaction that can be life-threatening.

All currently available epinephrine treatments require injection, which can be problematic for individuals with a fear of needles. Neffy, as a nasal spray, aimed to provide a needle-free alternative. During the advisory committee meeting, concerns were raised about the lack of data on Neffy’s performance in treating anaphylaxis, making it essential to ensure its effectiveness, especially given the drug’s intended use as an emergency treatment.

The rejection underscores the challenges of conducting clinical trials for emergency treatments for life-threatening conditions like anaphylaxis, where randomized controlled trials with placebos would be unethical. The drug’s manufacturer, ARS Pharmaceuticals, intends to resubmit its application to the FDA in the first half of 2024 to address the agency’s request for additional data.

While the rejection is disappointing for those seeking a needle-free option for epinephrine, it highlights the importance of rigorous clinical testing to ensure the safety and effectiveness of such emergency treatments.