Q&A: Company seeks to expand neffy FDA approval to more pediatric patients

Key takeaways:

• Children as young as age 10 years are able to self-administer neffy 1 mg.
• ARS Pharmaceuticals is also working on nasal spray treatments for type 1 hypersensitivity reactions, including urticaria.

ARS Pharmaceuticals announced its submission of a supplemental new drug application to the FDA for neffy 1 mg for the treatment of type 1 allergic reactions in children who weigh between 33 lb and 66 lb.

This announcement comes after the FDA approved neffy epinephrine nasal spray for emergency treatment of type 1 allergic reactions, including anaphylaxis, in pediatric and adult individuals weighing at least 66 lb in early August.

Healio spoke with Richard Lowenthal, MS, MSEL (MBA), cofounder, president and CEO of ARS Pharmaceuticals, about the new FDA submission and what it entails for the future of neffy.

Healio: Can you tell us about the importance of receiving neffy FDA approval for the treatment of younger pediatric patients?

Lowenthal: If approved by the FDA, neffy 1 mg for children who weigh 33 lb to 66 lb will be an extremely important tool for younger school-aged children with severe allergic allergies because, as with older patients and their caregivers, the fear of needles is an extremely big barrier to treatment. Given that nearly half of all current epinephrine autoinjector prescriptions are for children, and more than half of those are for children weighing 33 lb to 66 lb, there is an obvious need for innovative treatments such as neffy 1 mg.

Healio: What separates neffy 1 mg from other treatments that are available right now?

Lowenthal: If approved by the FDA, there are several factors that will separate neffy 1 mg from current autoinjector treatments.

As previously mentioned, the biggest attribute of neffy 1 mg will be that it is needle-free, which should help eliminate fear of treatment for many patients and their caregivers. In addition to removing that barrier, being needle-free also removes the risk for needle-related accidents, such as accidentally injecting into the hand or fingers of a child or caregiver. We have seen data that tell us that accidental needle sticks with epinephrine injection devices occur approximately 3,500 times each year in the U.S., and more often in pediatric patients who weigh 33 lb to 66 lb. We believe that taking away the fear of needles as well as removing the risk for accidental needle injuries will be very well received.

We also believe that neffy’s ease of use will help ensure that more people get treated at the first sign of an anaphylactic event. As with the FDA approved neffy 2 mg, neffy 1 mg is simple to insert into the nose and press to deliver epinephrine almost instantly. In fact, we conducted human factor studies that showed that adults without knowledge of the disease or device — for example, a teacher, a babysitter or a grandparent — were able to successfully use the device by simply reading the instructions. These studies also showed that untrained children as young as age 10 years can easily use neffy with self-administration.

Finally, we believe that neffy’s small size, which can easily fit into a pocket, purse or backpack, as well as its temperature variability — meaning that it can be exposed to temperatures up to 122°F (50°C) and, if accidentally frozen, it can be thawed and administered — will be beneficial to families with children who have severe allergic allergies.

Healio: What should doctors know about neffy 1 mg?

Lowenthal: If approved, doctors need to know that neffy 1 mg provides the same lifesaving treatment, epinephrine, as the treatments on which their patients currently rely, with the added benefit of being needle-free, which should help eliminate fear of treatment for many patients and their caregivers.

Healio: Can you tell us about the potential timeline of filing the supplemental new drug application and when you are expecting to hear about the submission?

Lowenthal: We are in discussions with the FDA and will know more details about the timeline in the next few months.

Healio: What else do you have in the works in terms of neffy products?

Lowenthal: Our team is currently working on advancing our innovative nasal spray development program across multiple type 1 hypersensitivity reactions, including acute flares of urticaria.

Urticaria is a skin disorder driven by mast cell degranulation and histamine release, which causes itchy wheals (hives) and angioedema, or both. In the U.S., the annualized incidence is approximately 5 million cases (1.56 per 100,000), and approximately 40% of cases become chronic urticaria, which can last for several years before spontaneous remission occurs.

Healio: Do you have any other comments?

Lowenthal: We are committed to ensuring access and affordability for all patients who need neffy. Our robust neffyConnect program can guide patients and caregivers through topics such as copay savings, insurance coverage support, at-home delivery options, refill reminders and identifying affordable pricing.

For patients with commercial insurance, neffy is available for $25 for two single-use devices. Patients with a high deductible plan, no insurance coverage or with an insurance plan that does not cover neffy can expect to pay $199 out of pocket for two doses. Neffy will be available for free for patients who have exhausted all other options.

For more information, we encourage patients, caregivers and physicians to visit neffy.com.

Reference:

• ARS Pharmaceuticals submits sNDA to FDA for neffy 1 mg dose for pediatric patients with type I allergic reactions who weigh 15 to 30 kg (33-66 lbs). https://ir.ars-pharma.com/news-releases/news-release-details/ars-pharmaceuticals-submits-snda-fda-neffyr-1-mg-dose-pediatric. Published Sept. 9, 2024. Accessed Sept. 16, 2024.