European designation follows U.S. FDA Orphan Drug Designation previously granted for the same indication
Landmark KIDCARE trial demonstrated superiority of infliximab over standard retreatment in resistant Kawasaki disease
Paris, France — May 21, 2026 — ODDIFACT, a TechDev company pioneering the repurposing of proven medicines towards rare diseases through artificial intelligence, today announced that the European Commission, following a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), has granted Orphan Drug Designation for infliximab for the treatment of Kawasaki disease.
This European designation follows the Orphan Drug Designation previously granted by the U.S. Food and Drug Administration (FDA) for the same indication, establishing a strong transatlantic regulatory foundation for the development of infliximab as a treatment for Kawasaki disease. ODDIFACT is the sponsor of both orphan designations.
Kawasaki disease is a rare, acute vasculitis that predominantly affects children under five years of age. It is the leading cause of acquired heart disease in children in developed countries. Without timely treatment, approximately 25% of affected children develop coronary artery aneurysms, which can lead to long-term cardiac complications, myocardial infarction, and death. The current standard of care, intravenous immunoglobulin (IVIG), fails in 10–20% of patients, leaving a critical unmet medical need for effective second-line therapies.
“This EMA Orphan Drug Designation is a defining milestone for ODDIFACT’s mission to enable treatments for the patients who need them most,” said Pierre-Alexandre Teulié, Co-Founder and Chief Executive Officer of ODDIFACT. “With orphan designations now secured on both sides of the Atlantic, we are building the regulatory pathway to make infliximab a recognized treatment option for children with Kawasaki disease. We aim to accelerate access to life-changing treatments for rare disease patients by leveraging decades of real-world safety and efficacy data, rather than starting from scratch.”
Robust Clinical Evidence: The KIDCARE Trial
The clinical rationale for infliximab in Kawasaki disease is supported by the landmark KIDCARE (Kawasaki Disease Comparative Effectiveness) trial, a Phase III, randomized, multicenter comparative effectiveness study conducted across 30 hospitals in the United States. Published in The Lancet Child & Adolescent Health in 2021, the KIDCARE trial enrolled 103 children with IVIG-resistant Kawasaki disease and demonstrated that infliximab was associated with shorter duration of fever, reduced need for additional rescue therapy, less severe hemolytic anemia, and shorter hospitalization compared with a second IVIG infusion.
“The KIDCARE trial demonstrated that infliximab is a safe, well-tolerated, and effective treatment for children with IVIG-resistant Kawasaki disease. These findings represent years of rigorous clinical investigation and confirm what many of us in the field have long believed: that repurposing well-established, approved biologic therapies for rare pediatric conditions is not only scientifically sound — it is a moral imperative,” said Jane C. Burns, M.D., Professor of Pediatrics at the University of California San Diego and Rady Children’s Hospital, and Principal Investigator of the KIDCARE trial. “Orphan drug designations like this one are essential to incentivizing the development of new indications for existing drugs, and to ultimately bringing better treatments to children with rare diseases who have been waiting far too long.”
About Orphan Drug Designation
Orphan Drug Designation is granted by the EMA to medicines intended for the diagnosis, prevention, or treatment of life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 individuals in the European Union. The designation provides important regulatory and financial incentives, including reduced fees for regulatory activities, protocol assistance, and the potential for up to 10 years of market exclusivity upon marketing authorization. In the United States, FDA Orphan Drug Designation provides similar incentives, including seven years of market exclusivity, tax credits for clinical development costs, and eligibility for FDA orphan drug grants.
About Kawasaki Disease
Kawasaki disease is an acute febrile vasculitis of unknown etiology that predominantly affects infants and young children. It is the most common cause of acquired heart disease in children in developed countries. The disease causes inflammation of blood vessels throughout the body, with a particular predilection for the coronary arteries. Standard treatment with high-dose IVIG and aspirin reduces the rate of coronary artery abnormalities, but 10–20% of patients are resistant to initial IVIG therapy and remain at elevated risk for serious cardiac sequelae. There is currently no approved second-line therapy for IVIG-resistant Kawasaki disease in either the United States or the European Union.
About Infliximab
Infliximab is a chimeric monoclonal antibody that binds to tumor necrosis factor alpha (TNF-α), a key pro-inflammatory cytokine involved in the pathogenesis of Kawasaki disease. Originally approved for the treatment of autoimmune and inflammatory conditions including rheumatoid arthritis, Crohn’s disease, and ulcerative colitis, infliximab has been used off-label in Kawasaki disease for over a decade. The KIDCARE trial provided the first high-quality, randomized evidence supporting its use in IVIG-resistant Kawasaki disease.
About ODDIFACT
ODDIFACT is a mission driven Tech Dev company dedicated to transforming the treatment landscape for rare diseases through the repurposing of proven, existing medicines. Leveraging proprietary artificial intelligence and deep medical expertise, ODDIFACT identifies untapped therapeutic potential in drugs that have already demonstrated safety and efficacy in other indications — potentially saving years of development time and dramatically reducing the cost and risk of bringing treatments to patients with rare conditions. In 2024 and 2025, ODDIFACT received more FDA Orphan Drug Designations than any other biopharmaceutical company worldwide. Co-founded by Pierre-Alexandre Teulié, who was personally diagnosed with a rare disease, the company is driven by a deeply held conviction that no patient should be left without treatment options simply because their condition is rare.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of applicable securities laws. These statements involve known and unknown risks, uncertainties, and other factors that may cause actual results to differ materially from those expressed or implied. Orphan Drug Designation does not constitute approval of a drug for any indication and does not guarantee regulatory approval. ODDIFACT undertakes no obligation to publicly update any forward-looking statements.
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