ARS Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for neffy® (epinephrine nasal spray) in the treatment of Allergic Reactions (Type I), including anaphylaxis for adults and children =30 kg. ARS Pharma plans to submit a Formal Dispute Resolution Request (FDRR) to appeal the issuance of this CRL. In the letter, the FDA requested completion of a pharmacokinetic/pharmacodynamic study assessing repeat doses of neffy compared to repeat doses of an epinephrine injection product under allergen-induced allergic rhinitis conditions to support approval.

This request comes after the recommendation of the FDA Advisory Committee (PADAC) in May 2023 to approve neffy without the need for additional studies to demonstrate its efficacy or safety. Further, FDA and ARS Pharma previously aligned in August 2023 on final physician?s labeling and a post-marketing requirement to conduct this study as informative for labeling. The PADAC meeting was held on May 11, 2023, and concluded a favorable benefit-risk profile of neffy, with a 16:6 vote in favor for adults and 17:5 vote in favor for children (=30 kg) for the treatment of patients with allergic reactions (Type I), including anaphylaxis.

In that session, no member of the Committee raised specific concerns about the result of the completed study in people with allergen-induced acute rhinitis with single-dose neffy, which showed enhanced absorption during the time period when a clinical response would be expected. As ARS Pharma previously agreed with FDA to conduct a repeat-dose study under allergen-induced allergic rhinitis conditions as a post-marketing commitment, ARS Pharma anticipates a resubmission to the FDA in the first half of 2024, positioning ARS Pharma for an anticipated FDA action date in the second half of 2024. ARS Pharma expects to have anticipated cash, cash equivalents and short-term investments on hand of approximately $195 million at the time of the anticipated launch of neffy, if approved in the second half of 2024.

The CRL requested additional information on nitrosamine impurities to be tested for based on new draft guidance issued after the neffy NDA submission. ARS Pharma does not believe the additional testing would be a rate-limiting step for its resubmission to the FDA. A marketing authorization application for neffy is also under review by the European Medicines Agency with a Committee for Medicinal Products for Human Use opinion expected by year end 2023.

Submissions to other regulatory authorities in additional countries are planned for 2024. Type I severe allergic reactions are serious and potentially life-threatening events that can occur within minutes of exposure to an allergen and require immediate treatment with epinephrine, the only FDA-approved medication for these reactions. While epinephrine autoinjectors have been shown to be highly effective, there are well published limitations that result in many patients and caregivers delaying or not administering treatment in an emergency situation.

These limitations include fear of the needle, lack of portability, needle-related safety concerns, lack of reliability, and complexity of the devices. There are approximately 40 million people in the United States who experience Type I severe allergic reactions due to food, venom or insect stings. Of those, only 3.3 million currently have an active epinephrine autoinjector prescription, and of those, only half consistently carry their prescribed autoinjector.

Even if patients or caregivers carry an autoinjector, more than half either delay or do not administer the device when needed in an emergency.