The recent green light for younger patients underscores the drug’s potential to transform the standard of care for those grappling with EoE.
In a landmark decision, the US Food and Drug Administration (FDA) has extended the approval of Dupixent® (dupilumab), marking a significant milestone in the treatment landscape for pediatric patients battling eosinophilic esophagitis (EoE). This groundbreaking expansion now allows children aged one to 11 years, weighing at least 15kg, to benefit from this innovative therapeutic option.
Dupixent, developed by Sanofi and Regeneron Pharmaceuticals, initially gained FDA approval in May 2022 for patients aged 12 years and older, weighing at least 40kg. The recent green light for younger patients underscores the drug’s potential to transform the standard of care for those grappling with EoE, a condition known for its debilitating and progressive impact on a patient’s ability to eat.
Dr. George Yancopoulos, Board co-chair, President, and Chief Scientific Officer at Regeneron, expressed enthusiasm about this approval, emphasizing that Dupixent is now the “first and only treatment option for EoE patients aged one year and older, weighing at least 15kg.”
Dupixent, a fully human monoclonal antibody, stands out as a non-immunosuppressant. It works by inhibiting signaling in the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways. The drug’s efficacy and safety were rigorously evaluated in the two-part Phase III EoE KIDS trial, which played a pivotal role in securing this expanded FDA approval.
According to data provided by Regeneron Pharmaceuticals and Sanofi, the Phase III study demonstrated compelling results:
- At 16 weeks, an impressive 66 percent of children receiving a higher dose of Dupixent achieved histological disease remission, defined as ≤6 eosinophils/high power field, in comparison to a mere three percent for those on placebo.
- Histological remission was sustained at week 52, with 53 percent of children treated with Dupixent in both study parts demonstrating continued positive outcomes.
- Even children who switched to Dupixent from placebo in Part B of the study exhibited histological remission at week 52, further supporting the drug’s efficacy.
Addressing the unmet needs of pediatric patients, Dr. Naimish Patel, Head of Global Development, Immunology and Inflammation at Sanofi, acknowledged that despite existing treatment options, 40 percent of children under the age of 12 in the US with eosinophilic esophagitis continue to experience symptoms of this challenging disease.
The approval of Dupixent for pediatric patients not only expands treatment options but also represents a beacon of hope for those facing the significant challenges posed by EoE. With its ability to target the underlying type 2 inflammation contributing to the disease, Dupixent has the potential to revolutionize care for children, building on its established success in adults and adolescents with EoE.
Experts in the field, Kathy Zagaroli, Senior Director of Quality Control at Kiniksa Pharmaceuticals, and Tom Bujold, Senior Director of Quality Control at REGENXBIO, stressed the importance of industry collaboration in overcoming challenges related to bioburden and sterility testing for gene therapies. Their insights, featured in a recent article titled “Developing Micro QC for ATMPs,” underscore the need for increased harmonization within the industry.
As the medical community celebrates this approval, it is a reminder of the ongoing commitment to advancing treatments for rare and challenging conditions. Dupixent’s expanded approval for pediatric EoE patients represents a crucial step forward in addressing the unmet needs of this vulnerable population and offers renewed hope for improved quality of life.