sNDA Goes to FDA for neffy 1 mg for Young Children with Type 1 Allergic Reactions

ARS Pharmaceuticals has announced its submission to the US FDA of a supplemental new drug application (sNDA) for neffy (epinephrine nasal spray) 1 mg for the treatment of type 1 allergic reactions, including anaphylaxis, in children weighing between 15 and 30 kg. The submission follows by 1 month the agency’s approval on August 9, 2024, of neffy 2 mg for the same indication in adults and children weighing 30 kg or more.

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If approved, neffy 1 mg will be the only needle-free epinephrine treatment available for the younger, school-aged population.

“Fear of needles is a common reason children refuse treatment with auto-injectors,” Richard Lowenthal, cofounder, president, and CEO of ARS Pharmaceuticals, said in the company’s press release. In the case of emergency treatment for severe allergic reactions that is a significant barrier, given that “Nearly half of all current epinephrine auto-injector prescriptions are for children, and more than half of those are for children weighing 15 to 30 kg (33-66 lbs), Lowenthal added.

More than 40% of the 6 million US children with food allergies have experienced an anaphylactic-like allergic reaction, according to the company’s press statement. Research shows, however, that needles frighten 2 out of 3 children and more than 50% of parents self-report fear about using their child’s potentially life-saving epinephrine autoinjector. Approval of neffy 1 mg would be “the first new delivery method for [young children] in more than 35 years,” Lowenthal said.

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In clinical trials, pharmacokinetic (PK) data for neffy 1mg were slightly higher than that of adults who receive the same dose and pharmacodynamic (PD) response was similar, according to the statement. Among the data submitted to the FDA were the following highlights:

• No risk of needle-related adverse events including accidental injection into the hand or fingers of a child or caregiver, an accident that occurs approximately 3500 time a year in the US with epinephrine injection devices and disproportionately affects children in the 15 to 30 kg weight range.
• Simple insert and press mechanism delivers epinephrine almost instantly without any hold time required in the nose.
• Human factor studies showed that adults without knowledge of the disease or device (eg, babysitter, teacher), were able to use it successfully by reading the instructions. Untrained children as young as 10 years of age were also able to easily self-administer neffy 1 mg.
• neffy allows for temperature exposure up to 122°F (50°C). If accidentally frozen, it can be thawed and administered.

Type I severe allergic reactions can occur within minutes of exposure to an allergen and require immediate treatment with epinephrine, the only FDA-approved medication for these often-life-threatening reactions, according to the news release. Evidence shows that epinephrine autoinjectors are highly effective but is countered by research demonstrating limitations that lead to delay of administration or not administering the treatment at all in in an emergency. Needle phobia, poor portability, needle-related safety concerns, lack of reliability, and complexity of the devices are among those limitations, ARS Pharmaceuticals said.

Evidence shows that epinephrine autoinjectors are highly effective but is countered by research demonstrating limitations that lead to delay of administration or not administering the treatment at all in in an emergency.

Type I severe allergic reactions due to food, venom or insect stings affect 40 million people in the US of whom it is estimated only 3.3 million currently have an active prescription for an epinephrine autoinjector prescription. Moreover, only half of that group carry their prescribed autoinjector consistently. Even more dismaying, more than half of people with type 1 allergic reactions or caregivers of children with the allergy delay or do not administer the device when needed.

“There is no doubt that this innovation is going to save lives. Patients need options, and neffy is helping to solve that issue,” Eleanor Garrow-Holding, president and CEO of the Food Allergy & Anaphylaxis Connection Team (FAACT), said in the news release. FAACT is a national patient advocacy group raising awareness for all individuals affected by food allergies and life-threatening anaphylaxis. “There has been little advancement in emergency treatments for this community, especially to address concerns about children carrying and using an epinephrine auto-injector. For the safety of our children with food allergies, making neffy 1mg available as soon as possible must be a high priority.”

Source: ARS Pharmaceuticals submits sNDA to FDA for neffy 1 mg dose for pediatric patients with type 1 allergic reactions who weigh 15 to 30 kg (33-66 lbs). News release. ARS Pharmaceuticals. September 9, 2024. Accessed September 11, 2024.