FDA approves dupilumab for children with eosinophilic esophagitis
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Key takeaways:
- Dupilumab is the first drug approved for treating children with eosinophilic esophagitis.
- The safety profile in children was similar to the safety profiles for adolescents and adults.
The FDA has approved the use of dupilumab for treating eosinophilic esophagitis in children aged 1 to 11 years who weigh at least 15 kg, according to a press release.
The approval expands the use of dupilumab (Dupixent; Regeneron, Sanofi) beyond adults and adolescents aged 12 years and older who weigh at least 40 kg.
Regeneron and Sanofi report that this approval came after an FDA Priority Review, indicating its potential for significant improvements in efficacy or safety in treating serious conditions.
Also, the companies say that dupilumab is now the first and only medicine approved in the United States for treating children with EoE.
“Young children are some of the most vulnerable patients with [EoE], as this debilitating and progressive disease threatens their basic ability to eat,” George D. Yancopoulos, MD, PhD, board co-chair, president and chief scientific officer at Regeneron, as well as a principal inventor of dupilumab, said in the press release.
About 21,000 children in the United States are using unapproved therapies for EoE that fail to target the root cause of their disease, the companies said, adding that the prevalence of the disease likely is higher since symptoms can be mistaken for other conditions and there may be delays in diagnosis.
The FDA approval was based on data from the phase 3 EoE KIDS trial of dupilumab’s use in children aged 1 to 11 years with EoE. At 16 weeks, 66% of children who received higher doses of dupilumab (n = 32) and 3% of those who received placebo (n = 29) achieved histological disease remission, defined as 6 or fewer eosinophils per high power field.
Also, 17 (53%) of the 32 children who received dupilumab experienced sustained remission at week 52. Eight (53%) of the 15 children who switched from placebo to dupilumab at week 16 experienced sustained remission at week 52 as well.
Further, the children in the dupilumab group had a greater decrease in the proportion of days where they experienced one or more signs of EoE based on the caregiver version of the Pediatric EoE Sign/Symptom Questionnaire from baseline through 16 weeks.
The safety profile through 16 weeks resembled the safety profiles for adolescents and adults using dupilumab through 24 weeks as well, the companies said. The most common adverse events included injection site reactions, upper respiratory tract infections, arthralgia and herpes viral infections.
Perspective
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The approval of dupilumab for children starting at age 1 year is an amazing advance for patients and families suffering from EoE. The scientific basis for using dupilumab for EoE is based in part by research conducted by my laboratory.
I am grateful to the CURED Foundation and NIH and others for their long-term financial support, which provided critical funds that were leveraged for the basic research that led to this important advancement. I am also grateful to Regeneron and Sanofi for pursuing the treatment of EoE, a relatively rare allergic disease with continual unmet need.
Our clinical studies have shown that the most common age for developing EoE in childhood is during the first 2 years of life. Having an effective medicine, such as dupilumab, available to be able to now treat young children is a long-awaited development in the field.
Reference:
Rothenberg E. J Allergy Clin Immunol. 2022;doi:10.1016/j.jaci.2022.09.027.
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