The U.S. Food and Drug Administration (FDA) has recently approved several new therapies aimed at addressing significant unmet needs in pediatric healthcare. These approvals span a range of conditions, including eosinophilic esophagitis, anaphylaxis, and atopic dermatitis, offering new treatment options for young patients and their families.
Dupilumab for Eosinophilic Esophagitis
Dupilumab, marketed as Dupixent by Regeneron Pharmaceuticals and Sanofi, received FDA approval on January 25, 2024, as the first treatment specifically indicated for children aged 1 to 11 years with eosinophilic esophagitis (EoE) who weigh at least 33 lb (15 kg). EoE is a chronic, progressive disease driven by type 2 inflammation that damages the esophagus. This approval addresses a critical unmet need in treating young children with this condition.
The approval was based on data from the Phase 3 EoE KIDS trial, which evaluated the safety and efficacy of dupilumab in children aged 1 to 11 years with EoE. The study demonstrated that 66% of children who received a higher, weight-tiered dose of dupilumab achieved histological disease remission, the study's primary endpoint, compared to only 3% of patients who received placebo (P < 0.0001).
According to Dr. Theresa Bingemann, Associate Professor of Pediatrics and Medicine at the University of Rochester School of Medicine and Dentistry, this approval "provides another option for patients who are still symptomatic on current treatments" and "gives families more options to decide what treatment fits best with their goals and values."
Epinephrine Nasal Spray for Anaphylaxis
ARS Pharmaceuticals' Neffy, an epinephrine nasal spray, was approved by the FDA on August 9, 2024, for the treatment of type 1 allergic reactions, including anaphylaxis, in adult and pediatric patients weighing at least 66 lb (30 kg). This marks the first nasal spray formulation of epinephrine approved for this life-threatening condition.
The approval was based on data demonstrating comparable epinephrine blood concentrations following administration of Neffy or approved epinephrine injection products in studies involving 175 healthy adults without anaphylaxis. Similar increases in blood pressure and heart rate were observed for Neffy compared with injection products, indicating comparable efficacy.
"Anaphylaxis is life-threatening, and some people, particularly children, may delay or avoid treatment due to fear of injections," said Dr. Kelly Stone, Associate Director in the Division of Pulmonology, Allergy, and Critical Care at the FDA’s Center for Drug Evaluation and Research. "The availability of epinephrine nasal spray may reduce barriers to rapid treatment of anaphylaxis."
Roflumilast Cream for Atopic Dermatitis
Roflumilast cream 0.15%, marketed as Zoryve by Arcutis Biotherapeutics, received FDA approval on July 9, 2024, for the treatment of mild to moderate atopic dermatitis (AD) in adults and children aged 6 years and older. This nonsteroidal topical treatment offers a new option for managing this common skin condition.
The FDA's decision was supported by positive results from the INTEGUMENT-1 and INTEGUMENT-2 trials, two identical Phase 3, parallel-group, double-blind, vehicle-controlled studies. These trials involved over 1300 adults and children aged 6 years or older with mild to moderate AD. Both studies met the primary endpoint of Investigator’s Global Assessment (IGA) success, defined as a Validated IGA-AD score of clear (0) or almost clear (1) plus a 2-grade improvement from baseline at week 4 (INTEGUMENT-1: 32% roflumilast cream vs 15.2% vehicle [P < .0001]; INTEGUMENT-2: 28.9% roflumilast cream vs 12% vehicle [P < .0001]).
Additionally, more than 40% of adults and children aged 6 years and older treated with roflumilast cream achieved a 75% reduction according to the Eczema Area and Severity Index at week 4 (INTEGUMENT-1: 43.2% vs 22% [P < .0001]; INTEGUMENT-2: 42% vs 19.7% [P < .0001]).
Dr. Lawrence Eichenfield, Professor of Dermatology and Pediatrics at the University of California, San Diego, noted that he is "pretty excited to get a new nonsteroidal medicine that we can put in our regimens of care."
