FDA Approves First Oral Anticoagulant for Children

Marcia Frellick

June 22, 2021

The US Food and Drug Administration (FDA) has approved dabigatran etexilate (Pradaxa) oral pellets for the treatment of venous thromboembolism (VTE) in children aged 3 months to less than 12 years. The drug is to be administered immediately after patients have completed a 5-day course of an anticoagulant given by injection.

The FDA also approved dabigatran etexilate oral pellets to prevent the recurrence of clots among patients in the same age group who have completed treatment for their first VTE.

With Monday's announcement, dabigatran etexilate becomes the first oral FDA-approved blood-thinning medication for children. The only other drug requires injection.

The drug was also approved in capsule form to treat blood clots in patients aged 8 years and older with VTE immediately after they have undergone at least 5 days of treatment with a blood thinner that was administered via injection and to prevent the recurrence of clots in patients 8 years and older who have completed treatment for their first VTE.

Ann Farrell, MD, director of the Division of Non-Malignant Hematology in the FDA's Center for Drug Evaluation and Research, said in a statement: "The FDA is committed to helping our youngest patients with serious medical conditions have treatments that are relatively easy to take."

The most common side effects include digestive system symptoms and bleeding. Dabigatran etexilate can cause serious and fatal bleeding and is not recommended for patients with bioprosthetic heart valves or triple-positive antiphospholipid syndrome.

A boxed warning cautions that stopping treatment early may increase the risk for clots and that spinal or epidural hematomas in patients undergoing spinal procedures may cause serious side effects.

Dabigatran etexilate was originally approved in 2010 for adults with nonvalvular atrial fibrillation to reduce risk for stroke and systemic embolism.

The drug's safety and efficacy for treating blood clots in patients younger than 18 years were evaluated in an open-label study of 267 pediatric patients. Patients were randomly assigned to receive either dabigatran etexilate or standard of care. The study compared the two groups for the number of patients who met the composite endpoint, which meant that they had not died from a blood clot, their blood clots had completely resolved, and they had no additional clots.

Researchers found that 81 (45.8%) of the 177 people who received the study drug met the composite endpoint, compared with 38 (42.2%) of the 90 patients who received standard of care.

The safety of dabigatran etexilate for the prevention of recurrence of blood clots in patients of the same age was evaluated in an open-label, single-arm study in 214 patients with a history of blood clots.

The primary endpoints were recurrence of blood clots, major and minor bleeding events, and death (both overall and related to blood clots). The long-term safety of dabigatran etexilate was similar to that found in the 267-patient study. Blood clots recurred in three patients, which was comparable to standard-of-care treatments.

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