Janssen Submits NDA for Use of Xarelto in Children

The Janssen Pharmaceutical Companies of Johnson & Johnson has submitted a New Drug Application (NDA) to the FDA for the use of Xarelto (rivaroxaban) in pediatric patients. The NDA seeks two pediatric indications: treatment of venous thromboembolism (VTE) and reduction in the risk of recurrent VTE in patients aged birth to less than 18 years of age; and thromboprophylaxis in patients aged 2 years and older with congenital heart disease who have undergone the Fontan procedure.

If approved, Xarelto will be the first oral Factor Xa inhibitor indicated in the United States for use in pediatric patients.

“The filing of this application is an important step in helping to address the burden of blood clots and provide doctors with optimal body weight-based dosing options in pediatric patients,” James List, M.D., Ph.D., global therapeutic area head, cardiovascular & metabolism, Janssen Research & Development, said in a statement.

Xarelto would be dosed based on body weight, either with an oral suspension formulation or tablets. The oral suspension formulation would be administered through a color-coded dosing device that was designed to help minimize dosing errors.

The application is based on data from studies of Xarelto in adults, as well as data from two phase 3 clinical trials in pediatric populations: EINSTEIN-Jr, which examined pediatric patients with previously diagnosed VTE, and UNIVERSE, which evaluated pediatric patients who are at risk of VTE after recently undergoing the Fontan procedure.

The EINSTEIN-Jr trial, published in the January 2020 issue of The Lancet Haematology, found that children treated with rivaroxaban had a low recurrence risk and reduced thrombotic burden without increased bleeding compared with standard anticoagulants. The study assessed 500 children, aged birth to 17 years, with previously diagnosed acute VTE who had started parenteral anticoagulation therapy.

The UNIVERSE trial is a two-part phase 3 trial examining children aged two to eight years who had the Fontan procedure, which is used for children born with congenital heart disease that redirects blood flow from the lower body to the lungs. Thrombosis can be a complication of this procedure.