Positive results were announced from a phase 3 trial evaluating the efficacy and safety of dupilumab in children aged 6 months to 5 years with moderate to severe atopic dermatitis.
The randomized, double-blind, placebo-controlled LIBERTY AD PRESCHOOL trial (ClinicalTrials.gov Identifier: NCT03346434) compared dupilumab with placebo, in addition to standard of care low potency topical corticosteroids, in 162 pediatric patients with uncontrolled moderate to severe atopic dermatitis. Patients were randomly assigned to receive dupilumab 200mg, 300mg, or placebo every 4 weeks based on body weight, in addition to topical corticosteroids.
The primary endpoints were the proportion of patients achieving an Investigator’s Global Assessment (IGA) score of 0 (clear) or 1 (almost clear) at week 16, and the proportion of patients with greater than or equal to 75% improvement from baseline in the Eczema Area and Severity Index (EASI 75) score at week 16.
Results demonstrated that after 16 weeks, 28% of patients in the dupilumab arm achieved an IGA score of 0/1 compared with 4% in the placebo arm (P <.0001). Additionally, 53% of patients treated with dupilumab achieved EASI 75 compared with 11% in the placebo arm (P <.0001). Treatment with dupilumab also met all key secondary endpoints vs placebo including:
- 70% average improvement from baseline in overall disease severity (EASI) vs 20% in the placebo arm (P <.0001);
- 49% average improvement from baseline in itch vs 2% in the placebo arm (P <.0001);
- Significant improvement in measures of observed patient outcomes (eg, sleep, skin pain, health-related quality of life), along with caregiver-reported health-related quality of life.
The safety profile of dupilumab in children was consistent with its known profile in atopic dermatitis. The most common adverse reactions for dupilumab and placebo were nasopharyngitis (8% vs 9%), upper respiratory tract infection (6% vs 8%), conjunctivitis (5% vs 0%), herpes viral infections (6% vs 5%), and injection site reactions (2% vs 3%), respectively. Detailed trial results will be presented at a future medical meeting.
“Currently, the standard of care for this patient population is topical steroids and other immunosuppressive medicines may be used which can damage delicate skin and, if used long-term, potentially impact growth,” said John Reed, MD, PhD, Global Head of Research and Development at Sanofi. “Knowing that safety is of the utmost importance for physicians and parents when considering treatment options for children and infants, we are encouraged by the results of this trial showing [dupilumab] addressed the signs and symptoms of atopic dermatitis without broadly suppressing the immune system, demonstrating the potential it could have for these very young patients.”
Dupilumab, an interleukin-4 receptor alpha antagonist, is marketed under the trade name Dupixent and is currently approved for the treatment of moderate to severe atopic dermatitis in patients 6 years of age and older. It is also indicated as an add-on maintenance treatment in patients with moderate to severe asthma and for inadequately controlled chronic rhinosinusitis with nasal polyps.
Dupixent® (dupilumab) pivotal trial meets all primary and secondary endpoints becoming first biologic medicine to significantly reduce signs and symptoms of moderate-to-severe atopic dermatitis in children as young as 6 months. News release. Regeneron Pharmaceuticals, Inc. Accessed August 31, 2021. https://www.prnewswire.com/news-releases/dupixent-dupilumab-pivotal-trial-meets-all-primary-and-secondary-endpoints-becoming-first-biologic-medicine-to-significantly-reduce-signs-and-symptoms-of-moderate-to-severe-atopic-dermatitis-in-children-as-young-as-6-months-301364919.html.