Topline
The Food and Drug Administration on Tuesday declined to approve a nasal spray that would have been the first needle-free alternative to injectors like EpiPen for treating severe allergic reactions, a decision that “surprised” developer ARS Pharmaceuticals and could push back the anticipated treatment’s arrival until late next year.
Key Facts
The FDA asked ARS Pharmaceuticals to conduct another study on its epinephrine nasal spray, Neffy, before it could be approved, according to a statement released by the company late on Tuesday.
The rejection came despite FDA advisors voting in favor of the regulator approving the drug for use in both adults and children in May and ARS Pharmaceuticals said it had come to an agreement with the regulator in August to collect the requested data on the safety of repeated doses once Neffy was on the market.
While the FDA is not legally bound to follow the votes of its committees, it is rare for the agency to deviate from their recommendations.
CEO Richard Lowenthal said the company is “very surprised” and “deeply disappointed” by the FDA’s decision and its “late” change of heart to require the study before approval, particularly given the endorsement from the agency’s advisory committee.
ARS Pharmaceuticals said it will appeal the FDA’s decision and plans to resubmit its application in the first half of 2024.
This timeline would give the FDA an “action date” for a response at some point in the second half of next year, ARS Pharmaceuticals said.
Key Background
Epinephrine, also known as adrenaline, has been used to treat allergic reactions for more than 100 years. The medicine can be lifesaving for those experiencing the most severe form of allergic reaction, anaphylaxis, which can come on suddenly after exposure to allergens like peanuts or shellfish and can kill without treatment. All treatment options for anaphylaxis on the market are injectable—often autoinjector pens like EpiPens that give a fixed single dose—which can spark fear or discomfort in some patients who are afraid of needles, can be impractical to carry around and are often prohibitively expensive. The potential life-threatening nature of anaphylaxis complicates the study of new drugs significantly and Neffy has not actually been tested in people experiencing anaphylaxis. This was a point of dispute among the FDA advisory panel—which nevertheless endorsed the drug—and the company collected alternative data to compare the drug to the injectors on the market. Its trials in healthy people, animals and people having a rhinitis attack, a type of allergic reaction in the nose, suggested the spray was comparable to injectors.
Crucial Quote
“I’m shocked,” Dr. Zachary Rubin, an allergist at Oak Brook Allergists in Illinois, told NBC News when asked about the FDA’s decision. Rubin, who was not on the FDA’s advisory committee, said “there are no alternatives” to needles for severe allergic reactions right now. “You basically have epinephrine autoinjector devices, needle options, and people have been clamoring for years to get a needle-free option,” Rubin said.