Motohiro Ebisawa, MD, PhD: Phase 3 Findings on Neffy for Pediatric Patients At-Risk of Anaphylaxis

In an interview with the HCPLive editorial team, Motohiro Ebisawa, MD, PhD, discussed his team’s recent findings on the intranasal epinephrine treatment ‘neffy’ for severe allergic reactions presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) 2024 meeting.¹

Ebisawa serves as director of the Clinical Research Center, Allergy and Rheumatology at Sagamihara National Hospital in Japan, and has served as president of the World Allergy Organization. Additionally, he served as principal investigator of the new phase 3 study on neffy’s use among patients with severe allergies.

“In Japan, we have the PMDA, which has authority to approve new drugs developed by pharmaceutical companies,” Ebisawa explained. “Likewise, in the United States and also Europe, the ARS pharmaceutical company applied the new drug called neffy with pharmacokinetics and pharmacodynamics data, and that was almost equivalent to that current available adrenaline auto-injector, called the EpiPen. However, the PMDA further requested us to perform a kind of real clinical trial for patients who maydevelop mild anaphylaxis, in case neffy really works or not.”

In the US, the US Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for neffy in 2023. The hotly-anticipated epinephrine alternative was developed for those with Type 1 allergic reactions like anaphylaxis and could have been a non-needle alternative to the EpiPen were it not for the setback.

“Last summer, we conducted a phase 3 study, recruiting 15 pediatric and also adolescents,” Ebisawa said. “Severe food allergy patients who are challenged with causative food allergens in our hospital. We tried to recruit a total of 15 patients for the clinical trial and, based on the body weight, we divided the patient group into 2 groups. That is neffy, 1.0 milligrams and 2.0 milligrams. We found that neffy really worked like old injectable adrenaline, like EpiPen, in our study.”

Ebisawa was also asked about the safety profile of neffy in his team’s research.

“The patients reacted to the oral food challenge of those causative food allergens, such as peanuts, tree nuts, and other types of food allergens, and most of the reactions were caused by the oral food challenge,” Ebisawa said. “However, adding neffy for the purpose of treatment actually did not induce any severe adverse events related to the drug itself. So it was quite safe and also very effective for the treatment of anaphylaxis.”

Ebisawa’s team also reported that there had not been any patients who had required a second dose of epinephrine within 15 minutes following their doses.¹

"So usually with auto-injectable adrenaline or epinephrine, intramuscular is the route,” Ebisawa noted. “The patients and guardians really hate the needle injection. So this is a great, great advantage for neffy, to make this drug a much better format for the treatment of anaphylaxis.”

References

1. ^{Ebisawa M, Lowenthal R, Tanimotoneffy S, et al. Epinephrine nasal spray, Demonstrates a Positive Efficacy and Safety Profile for the Treatment of Allergic Reactions in Pediatric Patients at-Risk of Anaphylaxis: Phase 3 Study Results. The Journal of Allergy and Clinical Immunology. February 2024. https://www.jacionline.org/article/S0091-6749(23)02379-5/fulltext. Date accessed: February 25, 2024.}