December 08, 2025
3 min read
Key takeaways:
- In pediatric patients, Anaphylm produced a maximum concentration that “slightly exceeded” the one found in adults.
- Researchers reported transient rises in systolic and diastolic blood pressure and heart rate.
ORLANDO — Pharmacokinetic and pharmacodynamic data of Anaphylm sublingual epinephrine film in pediatric patients were similar to data reported in adults, according to phase 1 study results.
This study on Anaphylm (AQST-109, Aquestive Therapeutics) was presented at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting.
Data were derived from Greenhawt M, et al. Phase 1 evaluation of sublingual film: Pharmacokinetics/pharmacodynamics safety tolerability in at risk pediatric patients. Presented at: ACAAI Annual Scientific Meeting; Nov. 6-10, 2025; Orlando.
Matthew Greenhawt
“These findings support the pharmacokinetic (PK) comparability between pediatric and adult populations,” Matthew Greenhawt, MD, MBA, MSc, FACAAI, FAAAAI, chief medical officer of the Asthma and Allergy Foundation of America, said during his presentation.
In an open-label, single-treatment, multi-site phase 1 study, Greenhawt and colleagues assessed 32 pediatric patients aged 7 to 17 years (median age, 13 years; 62.5% boys) with a history of allergic reactions and at high risk for serious allergic reactions to determine the pharmacokinetics (PKs) and pharmacodynamics (PDs) of 12 mg Anaphylm the day after sublingual administration.
Included patients had to weigh at least 30 kg (median weight, 47.15 kg; median BMI, 18.7 kg/m2), according to Greenhawt.
“Anaphylaxis is a very prominent problem,” Greenhawt said. “Pediatric risk is estimated to be anywhere from one to 761 per 100,000 person-years.”
As Healio previously reported, allergen-related symptoms in adults resolved within 5 minutes after an oral allergen challenge with the Anaphylm sublingual epinephrine strip. Sublingual delivery of epinephrine also achieved PK and PD results that were comparable with manual intramuscular injection and autoinjectors in a study published in Annals of Allergy, Asthma & Immunology.
Further, Anaphylm demonstrated durability despite testing in extreme temperatures and other environmental conditions.
The FDA accepted Aquestive Therapeutics’ new drug application for its Anaphylm sublingual epinephrine film for treating anaphylaxis and other type 1 allergic reactions in June. The Prescription Drug User Fee Act target action date is Jan. 31, 2026.
Results
In the pediatric population, Anaphylm produced a geometric mean maximum concentration (Cmax) of 568.6 pg/mL, and Greenhawt noted that this “slightly exceeded” the Cmax of 470.2 pg/mL found in a separate study of adults.
Additionally, the median time to Cmax was 10 minutes in pediatric patients, which was shorter than the median time to Cmax of 12 minutes in the adult study.
“Both of those … are considerably faster than comparative reference drugs,” Greenhawt said.
Notably, Greenhawt highlighted that there was no statistical difference between the two populations in a cross-study comparison of PKs.
Switching to PD data, researchers reported transient rises in:
- systolic blood pressure (mean maximum effect [Emax], 15.4 mm Hg; median time to maximum effect [TEmax], 11 minutes; mean area under the effect curve from 0 to 60 minutes [AUEC0-60], 8.7 h*mm Hg);
- diastolic blood pressure (mean Emax, 9.8 mm Hg; median TEmax, 8 minutes; mean AUEC0-60, 3.6 h*mm Hg); and
- heart rate (mean Emax, 12.5 bpm; median TEmax, 12 minutes; mean AUEC0-60, 5.9 h*bpm).
“This is exactly what you would expect to see when you give epinephrine: rapid response,” Greenhawt said.
“One of the features of this drug in particular is that sometimes you see a small dip with intermuscular injection of epinephrine, the diastolic actually dips and then recovers, and that’s not seen with this product,” he continued.
Similar to the PK data, Greenhawt noted that the pediatric PD data were comparable to that of adults.
Safety
Within the pediatric population, 84.4% reported any drug-related treatment-emergent adverse event. Greenhawt said at least 5% of patients reported hypoesthesia (12.5%; n = 4), paresthesia (12.5%; n = 4), discomfort (12.5%; n = 4), pain (6.3%; n = 2), nausea (12.5%; n = 4), vomiting (9.4%; n = 3) and upper abdominal pain (6.3%; n = 2).
Greenhawt characterized these reactions as local and site-specific.
In addition to patient-reported adverse events, clinicians/principal investigators reported adverse events during routine oral month checks. There were 23 clinician-observed events, and the most common event was exfoliation (nine events), followed by erythema (six events), pallor (five events) and ulcer (three events).
A slightly higher proportion of events were asymptomatic vs. symptomatic (13 events [57%] vs. 10 events).
“All but one of the clinician-observed events were [asymptomatic or] mild in severity,” Greenhawt said. “Of the three ulcerative events, only one was symptomatic.
“The safety results were consistent with the adult population,” he added.
Sources/Disclosures
Source:
Greenhawt M, et al. Phase 1 evaluation of sublingual film: Pharmacokinetics/pharmacodynamics safety tolerability in at risk pediatric patients. Presented at: ACAAI Annual Scientific Meeting; Nov. 6-10, 2025; Orlando.
References:
Disclosures:
Greenhawt reports having employment with the Asthma and Allergy Foundation of America; having a consulting role with Aquestive; holding membership of physician/medical advisory boards for ALK-Abelló, Aquestive, Bryn Pharma, DBV Technologies, Genentech, Novartis, Prota Therapeutics and Takeda; having speaker roles with ARS Pharma and Genentech; having unpaid membership on the National Peanut Board and medical advisory board of the International Food Protein-Induced Enterocolitis Syndrome Association; holding membership in the Brighton Collaboration Criteria Vaccine Anaphylaxis 2.0 working group; serving as senior associate editor of Annals of Allergy, Asthma & Immunology; and serving as a member of the Allergy Immunology Joint Taskforce on Practice Parameters..
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