FDA Advisory Committee Votes in Support of Favorable Benefit-Risk Profile for neffy® (Intranasal (IN) Epinephrine) for the Treatment of Allergic Reactions (Type 1), Including Anaphylaxis
- FDA Advisory Committee Votes: neffy Data Support a Favorable Benefit-Risk Assessment in Adults (16:6 in Favor) and in Children <18 years of age and ≥30 kg (17:5 in Favor)
- If Approved, neffy Will Become the First Needle-Free Epinephrine Product for the Treatment of Severe Allergic Reactions
- FDA Approval Decision Anticipated Mid-2023
“We’d like to thank PADAC members for their robust scientific discussions and clinical perspectives. The committee’s thoughtful review of the data, and support of the potential for neffy to address significant unmet needs of the severe allergy community, if approved, is highly encouraging,” said
Adverse events in neffy clinical trials were generally mild in nature without any meaningful nasal irritation or pain, and no serious adverse events were reported in any clinical study.
The PADAC decision was based on a review of comprehensive data from clinical studies developed in agreement with the FDA, which support a positive risk-benefit profile for intranasal (IN) epinephrine safety and effectiveness, compared to epinephrine injection. The studies demonstrated and the Committee’s discussion supported that neffy shows:
- Comparable pharmacokinetic (PK) data (epinephrine levels in the blood) vs. intramuscular injection including the first 10 to 20 minutes when clinical response is observed
- Comparable or greater pharmacodynamic (PD)response (systolic blood pressure and heart rate) vs. intramuscular injection that is observed even at 1 minute after dosing of neffy
- Effective IN delivery of systemic epinephrine and PD response even with nasal congestion or runny nose (e.g., during allergic rhinitis or upper respiratory tract infection)
- Comparable safety to injection that is generally mild in nature without any meaningful nasal irritation or pain, without needle-related risks
Additionally, neffy’s sprayer device has been approved by FDA for drug delivery for six other products in
“Patients need options – with different administration methods – to facilitate actual epinephrine use in an emergency event,” said
While PADAC recommendations are non-binding, the FDA will consider this outcome in its review of the pending New Drug Application (NDA) for neffy. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date anticipated mid-2023. If approved, neffy would be the first non-injectable treatment available to patients with allergic reactions (Type 1) including anaphylaxis.
“Millions of people have been waiting decades for a new delivery method of epinephrine, and we are grateful to the community for sharing their personal experiences and illustrating the potential neffy has in changing lives. We continue discussions with private payers, pharmacy benefit managers, and retail pharmacies to ensure that if neffy receives FDA approval, we are ready to make this important new treatment modality widely available for those who need it,”
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 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 25 to 40 million people in
ARS is a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect themselves from severe allergic reactions that could lead to anaphylaxis. The Company is developing neffy® (also referred to as ARS-1), an intranasal epinephrine product in clinical development for patients and their caregivers 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.
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 anticipated timing for regulatory review decisions on neffy and the potential approval of neffy; the implications of the PADAC decision; ARS being ready to make neffy, if approved, widely available for those who need it; the estimated addressable patient population for neffy; 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 “anticipate,” “believe,” “plan,” “will,” “potential,” “may” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon ARS’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; the PADAC decision should not be relied on as an indication that neffy will ultimately be approved; the FDA is not bound by the PADAC decision or any of its recommendations and there are a number of instances where the FDA has voted against the recommendations of advisory committees; the PDUFA target action date may be delayed due to various factors outside ARS’s control; the FDA’s prior approval of neffy’s sprayer device for drug delivery for six other products should not be relied on as an indication that FDA will approve the sprayer device for the administration of neffy; the labelling for neffy, if approved; the scope, progress and expansion of developing and commercializing neffy, if approved, including the ability to enter into distribution arrangements and obtain favorable reimbursement; the size and growth of the market therefor and the rate and degree of market acceptance thereof vis-à-vis intramuscular injectable products; ARS’s ability to protect its intellectual property position; the impact of government laws and regulations; and ARS’s ability to execute its plans and strategies. 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 the company’s Annual Report on Form 10-K for the year ended
The forward-looking statements included in this press release are made only as of the date hereof. ARS assumes no obligation and does not intend to update these forward-looking statements, except as required by law.
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