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

(CNN) — The U.S. Food and Drug Administration on Tuesday did not approve a needle-free epinephrine for the 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 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 the FDA typically 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 Pharmaceuticals said that the FDA requested the company assess 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 a news release late Tuesday. “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 due to food, venom, insect stings and medicines. Studies show that needles can be daunting and lead to people to hesitate before using the lifesaving medicine, although it works best when given quickly. One study showed that while 3.3 million people have a prescription for an epinephrine autoinjector, only half consistently carry it with them.

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