FDA declines to approve needle-free epinephrine, asks drugmaker for more data

By Jen Christensen, CNN

(CNN) — The US Food and Drug Administration on Tuesday did not approve a needle-free epinephrine for treatment of severe allergic reactions, a move that drugmaker ARS Pharmaceuticals called surprising. Instead, the agency requested more information about the nasal spray, including a study of repeat doses, ARS said in a news release.

In May, an independent advisory committee to the FDA recommended that the agency approve the drug, called Neffy.

On Tuesday — the day by which an FDA decision was due — the agency issued a Complete Response Letter, which it usually sends when it has questions about data submitted by a company seeking regulatory approval of a product. Typically, such a letter will list the deficiencies in a company’s submission and give recommendations as to what actions are needed to get FDA approval. In these cases, the company does not have to do an entire resubmission.

ARS said the FDA requested an assessment of how repeat doses of the nasal spray would do, compared with the injection form of epinephrine.

“We are very surprised by this action and the late requirement at this time to change the repeat-dose study from a post-marketing requirement, which we had previously aligned on with FDA, to a pre-approval requirement, particularly given the positive Advisory Committee vote,” Richard Lowenthal, co-founder, president and CEO of ARS Pharmaceuticals, said in the news release. “We stand by the totality of the neffy data package generated in a comprehensive registration program that was aligned upon with FDA and believe strongly in the value neffy can provide for patients, families and caregivers living daily with severe allergic reactions.”

If approved, Neffy would be the first needle-free nasal spray epinephrine treatment for people with severe allergic reactions. About 40 million people in the US experience extreme allergic reactions, often to things like foods, venom, insect stings and medicines.

Studies have showed that although 3.3 million people have a prescription for a prefilled epinephrine autoinjector, only half consistently carry it with them.

Epinephrine has always come in the form of an injection. Although autoinjectors have made shots easier, even people who’ve had practice can hesitate to use them, often because they’re afraid of the needle or because they’re just not comfortable with the technology. But hesitation can equal death.

A survey conducted by the the Asthma and Allergy Foundation of America found that only 19% of adult patients felt “very confident” using an autoinjector. Of the parents who were surveyed, 90% said their child had had a severe allergic reaction, but nearly 3 out of 4 of those parents said their child did not receive epinephrine. About 42% instead used an antihistamine, which cannot reverse life-threatening symptoms like epinephrine can.

“A nasal spray would give people managing severe allergic reactions (anaphylaxis) more options,” Melanie Carver, chief mission officer of the Asthma and Allergy Foundation of America, said in an email. “Fear of epinephrine and needle phobia are significant reasons why people are hesitant or delay treatment for a condition that requires immediate action.”

Epinephrine has a long safety record, Carver said. Her association supports the scientific process for drug development but was disappointed with the FDA decision.

The CEO of the nonprofit group Food Allergy Research & Education (FARE), Sung Poblete, had also hoped there would be another option for people with severe allergies. She said a nasal spray would be a lot easier to carry, and a needle-free option may be less daunting for bystanders to use if someone near them is having a bad allergic reaction.

Poblete, a nurse, said she was on a plane once when someone had a severe allergic reaction. She went to the medical kit that every plane has; inside was a vial of epinephrine and a syringe.

“I’m standing there with someone who cannot breathe. And I’m trying to calculate the weight of the patient to see how much to give them, and I’m like, ‘oh, my God, it’s too much,’ ” she said. “I’d rather just give them something like Neffy and just shoot it in their nose. It would be so much simpler. And remember, I’m trained for this. Imagine what it would be like for someone without medical training. Needles can be particularly difficult.”

“As one of the more than 33 million Americans with life-threatening food allergies, I am frustrated by the FDA’s decision today,” Poblete said Wednesday. “Our community believed this innovation would finally come to the more than 10 percent of Americans with life-threatening food allergies, but instead, the FDA will force us to wait even longer.”

ARS said that it anticipates resubmitting the necessary data to the FDA in the first half of 2024 and that it could get approval from the FDA in the second half of 2024.

Another company, Aquestive Therapeutics, is working a version of the medicine that goes under the tongue. Carver said options like these would be good to have.

“Ultimately,” she said, “we want people to feel confident taking their medicine, and we want it to be as easy for them to carry and use it.”

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