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.)

Rhea-AI Summary

ARS Pharmaceuticals (Nasdaq: SPRY) has submitted a supplemental New Drug Application (sNDA) for neffy® 1 mg, a needle-free epinephrine treatment for Type I Allergic Reactions, including anaphylaxis, in children weighing 15 to 30 kg (33-66 lbs.). This follows the FDA approval of neffy (2 mg) on August 9, 2024, for adults and children weighing 30 kg or more. Key highlights include:

• No risk of needle-related adverse events
• Simple insert and press mechanism for instant delivery
• Easy to use by untrained adults and children as young as 10
• Temperature resistant up to 122°F (50°C)

If approved, neffy 1 mg will be the first needle-free epinephrine option for younger children in over 35 years, addressing the needs of approximately six million children with food allergies in the U.S.

Positive

• Submission of sNDA for neffy® 1 mg, expanding potential market to children weighing 15-30 kg
• First needle-free epinephrine treatment for younger school-aged children if approved
• Addresses a significant market with nearly half of current epinephrine auto-injector prescriptions for children
• Eliminates risk of needle-related adverse events, which affect ~3,500 cases annually in the U.S.
• Simple administration mechanism and usability by untrained individuals
• Higher temperature resistance compared to traditional auto-injectors

Negative

• Approval for neffy 1 mg is still pending FDA review
• Pharmacokinetic data for neffy 1mg slightly higher than adults receiving the same dose

Insights

Medical Research Analyst

The submission of an sNDA for neffy® 1 mg is a significant development in pediatric allergy treatment. This needle-free epinephrine nasal spray, if approved, could revolutionize anaphylaxis management for children weighing 15-30 kg. Key points:

• Addresses needle phobia: 66% of children fear needles, potentially improving treatment adherence.
• Targets a important market: Nearly half of epinephrine auto-injector prescriptions are for children, with over half for the 15-30 kg weight range.
• Safety profile: Eliminates risks associated with accidental needle injections, which affect ~3,500 cases annually in the U.S.
• Ease of use: Simple administration mechanism, potentially improving emergency response times.

While promising, FDA approval is still pending and long-term efficacy data in real-world settings will be important for widespread adoption.

Pediatric Allergist

The potential approval of neffy® 1 mg could be a game-changer in pediatric allergy management. As a clinician, I'm particularly impressed by:

• PK/PD data: Slightly higher pharmacokinetics and similar pharmacodynamics to adult dosing suggest effective and safe delivery.
• Temperature stability: Functionality at up to 122°F (50°C) and after freezing addresses common storage concerns.
• Usability: Human factor studies demonstrating successful use by untrained adults and children as young as 10 are promising for real-world emergency scenarios.

However, we must remain cautious until we see robust real-world data on efficacy and any potential nasal irritation or other side effects in this younger population. Overall, this innovation could significantly improve anaphylaxis management in school-aged children.

Market Research Analyst

The market implications of neffy® 1 mg are substantial:

• Target market: With 6 million children affected by food allergies in the U.S. and 40% having experienced severe reactions, the potential user base is significant.
• First-mover advantage: As the first needle-free option for younger children in over 35 years, ARS Pharmaceuticals could capture a substantial market share.
• Competitive edge: Addressing key concerns like needle phobia and ease of use could drive preference over traditional auto-injectors.
• Broader accessibility: Potential for increased adoption in schools and public spaces due to its user-friendly design.

While the market opportunity is clear, success will depend on pricing strategy, insurance coverage and education efforts to drive adoption among healthcare providers and caregivers.

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

The submission of neffy 1 mg sNDA follows FDA approval on August 9, 2024, of neffy (2 mg) for the treatment of Type I Allergic Reactions, including anaphylaxis, in adults and children who weigh 30 kg (66 lbs.) or greater

SAN DIEGO, Sept. 09, 2024 (GLOBE NEWSWIRE) --  ARS Pharmaceuticals, Inc. (Nasdaq: SPRY), a biopharmaceutical company dedicated to empowering at-risk patients and their caregivers to better protect patients from severe allergic reactions that could lead to anaphylaxis, announced today the submission of a supplemental New Drug Application (sNDA) for neffy^® 1 mg for the treatment of Type I Allergic Reactions, including anaphylaxis, in children who weigh 15 to 30 kg (33-66 lbs.). Pharmacokinetic (PK) data for neffy 1mg were slightly higher than that of adults who receive the same dose and pharmacodynamic (PD) response was similar.

Food allergies affect approximately six million children in the U.S., of which more than 40 percent have experienced a severe allergic reaction such as anaphylaxis. Additionally, studies show that two out of three children are afraid of needles and more than half of parents express fear about using their child’s epinephrine auto-injector – a potentially lifesaving treatment.

“Fear of needles is a common reason children refuse treatment with auto-injectors. If approved, neffy 1mg will be the first needle-free epinephrine option for younger children, in addition to being the first new delivery method for this population in more than 35 years,” says Richard Lowenthal, Co-Founder, President and CEO of ARS Pharmaceuticals. “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.). We believe there is a clear need for innovative treatments like neffy 1mg.”

Highlights from the data submitted to the FDA underscore several key attributes:

• No risk of needle-related adverse events including accidental injection into the hand or fingers of a child or caregiver. This occurs approximately 3,500 times each year in the U.S. with epinephrine injection devices and disproportionately affects 15 to 30 kg (33-66 lb.) pediatric patients.
• 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 (e.g. a babysitter or a teacher), were able to successfully use the device by simply reading the instructions. These studies also showed that untrained children as young as 10 years of age can easily use neffy with self-administration.
• neffy allows for temperature exposure up to 122°F (50°C). If accidentally frozen, it can be thawed and administered.

“There is no doubt that this innovation is going to save lives. Patients need options, and neffy is helping to solve that issue,” said Eleanor Garrow-Holding, President and CEO of the Food Allergy & Anaphylaxis Connection Team (FAACT), 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.”

About Type I Allergic Reactions including Anaphylaxis
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 auto-injectors 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 auto-injector prescription, and of those, only half consistently carry their prescribed auto-injector. Even if patients or caregivers carry an auto-injector, more than half either delay or do not administer the device when needed in an emergency.

About ARS Pharmaceuticals, Inc.
ARS Pharma is a biopharmaceutical company dedicated to empowering at-risk patients and their caregivers to better protect patients from severe allergic reactions that could lead to anaphylaxis. The Company is commercializing neffy^® 2mg (trade name EURneffy in the EU) (previously referred to as ARS-1), an epinephrine nasal spray for patients 30 kg or greater with Type I allergic reactions including food, medications and insect bites that could lead to life-threatening anaphylaxis. For more information, visit www.ars-pharma.com.

Forward-Looking Statements
Statements in this press release that are not purely historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include but are not limited to: the potential market and demand for neffy; the needle-free profile of neffy potentially increasing the likelihood that patients and caregivers may carry and administer epinephrine; neffy’s potential benefits to patients and caregivers; and other statements that are not historical fact. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “can,” “could,” “potential,” “will,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon ARS Pharma’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation: the ability to obtain and maintain regulatory approval for neffy; potential safety and other complications from neffy; the labelling for neffy in any future indication or patient population, if approved; the scope, progress and expansion of developing and commercializing neffy; the potential for payors to delay, limit or deny coverage for neffy; the size and growth of the market therefor and the rate and degree of market acceptance thereof vis-à-vis intramuscular injectable products; ARS Pharma’s ability to protect its intellectual property position; and the impact of government laws and regulations. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption “Risk Factors” in ARS Pharma’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, filed with the Securities and Exchange Commission (SEC) on August 6, 2024. This and other documents ARS Pharma files with the SEC can also be accessed on ARS Pharma’s website at ir.ars-pharma.com by clicking on the link “Financials & Filings” under the “Investors & Media” tab.

ARS Investor Contact:
Justin Chakma
ARS Pharmaceuticals
justinc@ars-pharma.com

ARS Media Contact:
Christy Curran
Sam Brown Inc.
615.414.8668
christycurran@sambrown.com

FAQ

What is the status of ARS Pharmaceuticals' neffy® 1 mg for pediatric patients?

ARS Pharmaceuticals (SPRY) has submitted a supplemental New Drug Application (sNDA) to the FDA for neffy® 1 mg, intended for pediatric patients weighing 15 to 30 kg (33-66 lbs.) with Type I Allergic Reactions, including anaphylaxis.

When was neffy® 2 mg approved by the FDA for SPRY?

The FDA approved neffy® 2 mg on August 9, 2024, for the treatment of Type I Allergic Reactions, including anaphylaxis, in adults and children weighing 30 kg (66 lbs.) or more.

What are the key advantages of neffy® 1 mg for pediatric patients?

neffy® 1 mg is needle-free, reducing the risk of needle-related adverse events. It features a simple insert and press mechanism, is easy to use by untrained individuals, and can withstand temperatures up to 122°F (50°C).

How many children in the U.S. could potentially benefit from SPRY's neffy® 1 mg?

Approximately six million children in the U.S. have food allergies, with more than 40 percent having experienced a severe allergic reaction such as anaphylaxis, indicating a significant potential market for neffy® 1 mg.