Sanofi and Regeneron’s Dupixent (dupilumab) has been approved by the US Food and Drug Administration (FDA) to treat eosinophilic oesophagitis (EoE) in patients aged one to 11 years and weighing at least 15kg.
The regulator’s decision expands its previous approval of the drug in patients aged 12 years and older and weighing at least 40kg.
EoE is a chronic inflammatory disease associated with type 2 inflammation that is thought to be responsible for damaging the oesophagus and impairing its function. Symptoms vary between patients, especially in those of different ages, but the condition is mostly characterised by vomiting, stomach or chest pain, and difficulty swallowing.
Approximately 21,000 children aged under 12 years in the US are currently being treated for EoE with unapproved therapies. However, the actual prevalence of children with the disease is likely higher, as symptoms can be mistaken for other conditions and there are delays in diagnosis.
Dupixent, which is now the first and only medicine approved in the US specifically indicated to treat young children with EoE, is a fully human monoclonal antibody that inhibits the signalling of the interleukin-4 and interleukin-13 pathways, shown to be central to type 2 inflammation.
The FDA’s latest decision was supported by positive results from the phase 3 EoE KIDS trial, in which 66% of children aged one to 11 years with EoE who received a higher dose of Dupixent at tiered dosing regimens based on weight achieved histological disease remission compared to 3% for placebo at week 16 – meeting the study’s primary endpoint.
Histological remission was sustained at week 52 in 53% of children treated with Dupixent in both parts of the trial and was achieved at week 52 in another 53% of those who switched to Dupixent from placebo in the second part.
George Yancopoulos, board co-chair, president and chief scientific officer at Regeneron, said: “Young children are some of the most vulnerable patients with EoE, as this debilitating and progressive disease threatens their basic ability to eat.
“Until today, these children had no approved treatment options specifically for EoE, leaving many with unapproved medicines that failed to target the root cause of their disease.”
Naimish Patel, head of global development, immunology and inflammation at Sanofi, added that the approval “brings hope to these patients who are at a critical age where struggling to eat and maintain weight directly impacts their overall nutritional intake and development”.