FDA rejects nasal epinephrine alternative

On Tuesday, Sept. 19, the Food and Drug Administration (FDA) rejected the approval of ARS Pharmaceutical’s product Neffy, an epinephrine nasal spray designed as an alternative to the conventional needle injection treatment. This decision surprised many because the Pulmonary Allergy Drug Committee of the FDA overwhelmingly accepted the drug at a meeting in May of 2023. 

Currently, there is no permanent treatment for food allergies. The primary approach is avoidance, with the use of epinephrine recommended in the case of anaphylaxis, the most severe reaction to the consumption of an allergen. 

The only current emergency treatment for accidental allergen exposure is the widely recognized epinephrine auto-injector (EpiPen). For people with severe food allergies, having an EpiPen on-hand is necessary for personal safety. 

An EpiPen contains a needle loaded with epinephrine that will relax the airway and intestinal muscles and increase blood pressure, giving the recipient time to get to a hospital and receive medical treatment. In the event of an allergic reaction, the person or someone nearby must inject the needle into the thigh for the epinephrine to enter the bloodstream.

Emergency treatment can be stressful and complicated. According to the NIH, the needed epinephrine dose in children having an allergic reaction is often withheld. “This reluctance to use needle-based injection devices in a timely manner is often driven by fear,” ARS Pharmaceutical president Robert Lowenthal said. Consequently, some parents fear the injection could be traumatic for their children and choose to rush them to the emergency room instead. 

ARS Phamaceutical’s drug Neffy represented an attempt to simplify dosage administration by employing a needle-free approach. The nasal spray aims to replace the traditional EpiPen and decrease the delay or absence of epinephrine use during a reaction. In 2021, between 25 and 40 million Americans experienced a severe allergic reaction while approximately 1.5 million regularly carry doses of epinephrine. This discrepancy explains the high hospitalization and fatality rates associated with food allergies. ARS Pharmaceutical marketed Neffy as a “better” treatment that would appeal to more people in emergencies. 

The medical world had high hopes that Neffy would be approved by the FDA on Sept. 19 after the treatment received positive feedback from the preliminary committees assigned to its case, in a 16-6 vote for use in adults and 17-5 in children. However, instead of approval, the FDA requested that ARS Pharmaceuticals conduct an additional study to substantiate their findings. 

“Our community believed this innovation would finally come to the aid of more than 10 percent of Americans with life-threatening food allergies, but instead, the FDA will force us to wait even longer,” Sung Poblete, the CEO of Food Allergy Research and Education (FARE), said.

Neffy began the early clinical development stages in 2020 and first presented trial data and conclusions at the American Academy of Allergy, Asthma and Immunology in 2023. Many physicians and specialists see this drug as necessary to better treat people with food allergies. 

“Seconds count when someone is experiencing anaphylaxis and any delay in treatment could have life-threatening consequences,” the Allergy & Asthma Institute said in a release. “A needle-free epinephrine option will address an unmet need – and it’s needed sooner rather than later.”

Questions posed by the FDA to ARS Pharmaceuticals revolve around their minimal amounts of concrete human trials. While trials on animals and people without anaphylaxis yield promising results, conducting allergen trials on those likely to experience anaphylaxis raises ethical concerns; epinephrine decreases in efficacy in the body over time, explaining the lack of relevant experimentation. 

“While I think the drug has merit, it doesn’t have adequate efficacy data at this point,” Dr. Maryann Amirshahi, doctor of emergency medicine, affirmed.

Article written by Samantha Cutler of Richard Montgomery Highschool 

Photo by Samantha Cutler of Richard Montgomery Highschool