Xarelto Becomes the First DOAC FDA-Approved for Use in Pediatric Patients After Fontan Procedure

The FDA has added two new pediatric indications for Xarelto (rivaroxaban). The regulatory agency’s decision in late December 2021 makes Xarelto the first FDA-approved direct oral anticoagulant (DOAC) for prevention venous thromboembolism (VTE) in patients ages 2 years and older with congenital heart disease after undergoing the Fontan procedure.

The FDA also gave the go-ahead for Xarelto as a treatment of VTE and to reduce the risk of recurring VTE in patients from birth to less than 18 years of age after receiving at least 5 days of initial parenteral anticoagulation therapy.

Xarelto is the only DOAC available in an oral suspension, which facilitates pediatric weight-based dosing.

The Fontan procedure is a heart surgery performed in pediatric patients with a single functional heart ventricle. The procedure allows blood to flow directly from the heart into the lungs and leaves the functioning ventricle open to pump blood to the rest of the body.

Thrombosis is one of the most common complications associated with the Fontan procedure, and up to 25% of these thromboembolic events can be fatal.

Xarelto, developed by Janssen Pharmaceuticals, is a selective factor Xa inhibitor that directly binds to factor Xa to prevent thrombin formation. Some of its other indications are as a treatment for pulmonary embolism, prophylaxis of venous thromboembolism and risk reduction of major thrombotic vascular events in people with peripheral artery disease.

The approval of the new indications was based on results from two Phase 3 trials. The UNIVERSE study enrolled 112 children ages 2 to 8 years with single ventricular function and who had undergone the Fontan procedure within four months before trial enrollment. Participants taking Xarelto had fewer thromboembolic events than those taking aspirin, although results were not statistically significant. Both groups had similar rates of adverse events. The EINSTEIN-Jr study enrolled 500 children ages birth to 17 years with previously diagnosed VTE and who had begun parenteral anticoagulation treatment. After 3 months, a smaller percentage of children taking Xarelto showed signs of recurrent VTE versus participants using standard anticoagulation therapy.