The U.S. Food and Drug Administration (FDA) has approved dabigatran etexilate oral pellets for the treatment of children ages three months to 12 years with venous thromboembolism (VTE) directly after receiving injectable anticoagulants for at least five days. The treatment also is approved as prophylaxis for recurrent thrombosis in patients ages three months to 12 years who have completed treatment for first VTE.
This approval is based on safety and efficacy data from a study of 267 pediatric patients who were randomly assigned to receive either dabigatran etexilate or standard of care. The primary composite endpoint consisted of the proportion of patients with complete thrombus resolution, as well as freedom from recurrent VTE and VTE-related death. Of the 177 patients treated with dabigatran etexilate, 81 (46%) met the composite endpoint, compared with 38 (42%) of 90 patients receiving standard therapy.
“The FDA is committed to helping our youngest patients with serious medical conditions have treatments that are relatively easy to take,” said Ann Farrell, MD, director of the division of non-malignant hematology in the FDA’s Center for Drug Evaluation and Research. “With today’s approval of [dabigatran etexilate], pediatric patients have another therapeutic option to treat and prevent potentially deadly blood clots.”
This indication includes a boxed warning cautioning that early treatment discontinuation may increase the risk of thrombosis, and that in patients undergoing spinal procedures, spinal or epidural hematomas may cause serious complications. As dabigatran etexilate can cause severe and fatal bleeding, it is not recommended for patients with bioprosthetic heart valves or triple-positive antiphospholipid syndrome.
Dabigatran etexilate is the first FDA-approved oral anticoagulant for children. In 2010, the medication was approved to reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation.
Sources: FDA press release, June 21, 2021; Halton J, Brandão LR, Luciani M, et al. Dabigatran etexilate for the treatment of acute venous thromboembolism in children (DIVERSITY): a randomised, controlled, open-label, phase 2b/3, non-inferiority trial. Lancet Haematol. 2021;8(1):e22-e33.