The Food and Drug Administration (FDA) has declined to grant approval for an epinephrine nasal spray, Neffy, which could have been a groundbreaking needle-free alternative to epinephrine autoinjectors like EpiPens. This decision, communicated to drug manufacturer ARS Pharmaceuticals, has come as a surprise to many, particularly since the FDA’s advisory committee had previously recommended the drug’s approval for both children and adults during a May vote. Such instances of the FDA not aligning with its advisory committees are relatively rare.
ARS Pharmaceuticals expressed its astonishment at the FDA’s action, with CEO Richard Lowenthal stating, “We are very surprised by this action.” The company intends to appeal the FDA’s request for additional data to support Neffy’s approval.
Epinephrine, a crucial treatment for reversing anaphylaxis, the most severe form of allergic reaction, has been in use in the United States since 1901. However, all existing epinephrine treatment options require injection, a hurdle for individuals with needle phobias.
Dr. Zachary Rubin, an allergist at Oak Brook Allergists in Illinois, expressed shock at the FDA’s decision, emphasizing the long-standing need for a needle-free alternative. He remarked, “You basically have epinephrine autoinjector devices, needle options, and people have been clamoring for years to get a needle-free option.”
During the advisory committee meeting in May, a major concern was the lack of clinical data, especially the absence of studies involving individuals experiencing anaphylaxis. ARS Pharmaceuticals contended that the nasal spray was “comparable” to an EpiPen, based on studies conducted in animals and non-anaphylactic individuals.
However, Dr. Maryann Amirshahi, a professor of emergency medicine at Georgetown University School of Medicine and a committee member, cited the absence of data in patients with anaphylaxis as a significant issue. She voted against the drug’s approval on both occasions, stating, “the scary thing was not the shot, but a drug failing in treating a life-threatening condition.”
The ethical challenges associated with studying a drug intended for the emergency treatment of life-threatening allergic reactions were also acknowledged. Conducting a randomized controlled trial involving patients with severe allergic reactions receiving either the treatment or a placebo would be unethical, as pointed out by Dr. Rubin.
Amirshahi suggested that the drug could potentially be studied in an allergist’s office or an emergency room where a fail-safe treatment is readily available. She expressed hope that the FDA’s rejection would allow for adequate time to conduct a comprehensive study of the drug’s effectiveness.
As part of its request for additional research, the FDA has asked for a study comparing Neffy to an epinephrine autoinjector in individuals with allergen-induced nasal symptoms, such as sneezing, itching, and congestion. ARS Pharmaceuticals intends to resubmit its application to the FDA in the first half of 2024.