Janssen Submits New Drug Application to FDA for Xarelto in Pediatric Patients

June 23, 2021—The Janssen Pharmaceutical Companies of Johnson & Johnson announced it has submitted a New Drug Application (NDA) to the FDA for the use of the oral Factor Xa inhibitor, Xarelto (rivaroxaban), in pediatric patients.

The NDA seeks two pediatric indications:

1. Treatment of venous thromboembolism (VTE) and reduction in the risk of recurrent VTE in patients aged birth to < 18 years after at least 5 days of initial parenteral anticoagulant treatment
2. Thromboprophylaxis in patients age ≥ 2 years with congenital heart disease who have undergone the Fontan procedure

According to Janssen, current guidelines are limited and recommend treating pediatric patients with or at risk for reoccurring blood clots with standard anticoagulant therapy, which often requires painful injections, dietary restrictions, and regular laboratory monitoring.

There are currently no FDA-approved anticoagulation therapies for pediatric patients with congenital heart disease who have undergone the Fontan procedure. The limited guidance for managing these patients leaves physicians to extrapolate adult data to infer pediatric dosing and then regularly monitor their patients, noted the company.

Janssen advised that the application is based on evidence from adequate and well-controlled studies of Xarelto in adults as well as data from two phase 3 clinical trials of Xarelto 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.

For both potential indications, 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.

Earlier in 2021, Janssen’s development partner Bayer received approval in Canada; the European Union, including the United Kingdom; Japan; and Switzerland for Xarelto for the treatment of VTE and prevention of VTE recurrence in children and adolescents aged < 18 years after at least 5 days of initial parenteral anticoagulation treatment, noted the Janssen announcement.