The FDA approved the first direct oral anticoagulant (DOAC) for the treatment of children with venous thromboembolism (VTE), the agency announced on Monday.
Approval for dabigatran (Pradaxa) stipulates that it be given to children after they have received a blood thinner injection for at least 5 days. The drug was also approved for preventing recurrent clots in children who have completed treatment for their first VTE.
Dabigatran comes in two age-appropriate formulations: pellets for children from 3 months to 12 years of age, and capsules for children age 8 years and older.
“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, of FDA’s Center for Drug Evaluation and Research. “With today’s approval of Pradaxa, pediatric patients have another therapeutic option to treat and prevent potentially deadly blood clots.”
Pediatric VTE may arise as a secondary complication of conditions such as a venous catheter, cancer, infection, congenital heart disease, trauma, or surgery. FDA had approved dalteparin (Fragmin), as a subcutaneous injection, to treat VTE in children in 2019.
Approval for dabigatran in pediatric patients was based on DIVERSITY, a 267-person open-label trial that included children up to 18 years of age. In this study, dabigatran (in various formulations) was found to be non-inferior to standard of care in terms of the composite primary endpoint of complete thrombus resolution, and freedom from recurrent VTE or death.
Further safety data came from a single-arm study of 214 children who needed further anticoagulation after completing initial treatment for confirmed VTE or after completing the DIVERSITY study.
Dabigatran activity may be reversed with a dedicated monoclonal antibody, idarucizumab (Praxbind). Another DOAC, rivaroxaban (Xarelto), had been shown to work as a treatment for blood clots in children.