First Oral Medication for Blood Thinning in Children Granted FDA Approval

Officials with the FDA have approved dabigatran etexilate (Pradaxa; Boehringer Ingelheim Pharmaceuticals, Inc) oral pellets for the treatment of children aged 3 months to less than 12 years of age with venous thromboembolism. The therapy is indicated for administration directly after these patients have been treated with a blood thinner given by injection for at least 5 days.

The FDA also approved dabigatran etexilate oral pellets for the prevention of recurrent clots in patients aged 3 months to less than 12 years of age who have completed treatment for their first venous thromboembolism. Additionally, dabigatran etexilate was approved in capsule form to treat blood clots in patients 8 years of age and older with venous thromboembolism directly after they have been treated with a blood thinner given by injection for at least 5 days, and to prevent recurrent clots in patients 8 years of age and older who have completed treatment for their first venous thromboembolism.

According to the FDA, dabigatran etexilate is the first approved medication for blood thinning that children can take orally. The only other approved blood thinning medication for children is given by injection.

Dabigatran etexilate was originally approved in 2010 to reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation.

According to the FDA, blood clots can pose a serious problem in children and adults. Children are most at risk for blood clots if they have cancer, congenital heart disease, a central venous catheter, or are admitted to an intensive care unit. Venous thromboembolism can lead to complications, including swelling and discomfort near the clot, chest pain, lung damage, anddeath.

The safety and efficacy of dabigatran etexilate as a treatment for blood clots in patients younger than 18 years of age was evaluated in a study of 267 pediatric patients. In this open-label study, patients were randomly assigned to receive either dabigatran etexilate or standard of care. The study compared the 2 groups for the number of patients who met the composite endpoint, having survived a blood clot, having their blood clots completely resolved, and having no additional blood clots. Results showed that 81 of the 177 people taking dabigatran etexilate met the composite endpoint compared to 38 of the 90 patients who received standard of care.

The safety of dabigatran etexilate to prevent recurrent blood clots in the same pediatric population was evaluated in an open-label, single-arm study in 214 patients with a history of blood clots. The primary endpoints of the study were recurrence of blood clots, major and minor bleeding events, and death, overall and related to blood clots. Recurrence of blood clots occurred in 3 of 214 patients, which was comparable to prior standard-of-care treatments.

The most common adverse effects (AEs) of dabigatran etexilate include digestive system symptoms and bleeding. This drug can cause serious and fatal bleeding, and it is not recommended for patients with bioprosthetic heart valves or triple-positive antiphospholipid syndrome. Additionally, dabigatran etexilate has a boxed warning cautioning that early treatment discontinuation may increase the risk of blood clots and that blood accumulation within parts of the spinal cord in patients undergoing spinal procedures may cause serious AEs.

REFERENCE

FDA Approves First Oral Blood Thinning Medication for Children [news release]. June 21, 2021; FDA. Accessed June 22, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-blood-thinning-medication-children