FDA approves drug to treat, help prevent types of blood clots in certain pediatric populations

Action

FDA has approved Xarelto (rivaroxaban) as tablets and an oral suspension to treat venous thromboembolism (VTE), or blood clots that form in the veins, and reduce the risk of VTE recurring in pediatric patients from birth to younger than 18 years who have received at least five days of injectable or intravenous treatment for blood clots.

Today, FDA has also approved Xarelto to prevent blood clots in pediatric patients two years and older with congenital (present from birth) heart disease after the Fontan procedure, a type of open-heart surgery. 

FDA approved Xarelto in tablet form in 2011; Xarelto as an oral suspension is a new dosage form the agency approved today. FDA previously approved Xarelto to treat, prevent, or reduce the risk of various blood clotting conditions in certain patient populations and for other uses.

Disease or Condition

Blood clots can be serious and develop in children. Risk factors for blood clots include central venous catheters, cancer, infection, surgery, and congenital heart disease. In addition, blood clots can develop after the Fontan surgical procedure for congenital heart disease. Without treatment, blood clots can restrict or block blood flow and oxygen, which can damage the body’s tissues or organs and can result in death.

Effectiveness

The effectiveness of Xarelto to treat and reduce the risk of recurrent VTE was studied in pediatric patients with confirmed VTE. After an initial period, 500 pediatric patients were randomly assigned to receive either Xarelto or standard-of-care medications for three months (or one month for children younger than two years with a central venous catheter related-VTE). At the end of the study, 1.2% of patients in the Xarelto treatment group had signs or symptoms of recurrent VTE, compared with 3.0% of patients in the standard-of-care group.

The effectiveness of Xarelto to prevent blood clots after the Fontan procedure was studied in pediatric patients between ages two and eight years with congenital heart disease who had a recent Fontan surgical procedure. In Part A of the study, patients received Xarelto for one year; in Part B, patients were randomly assigned to receive Xarelto or aspirin for one year. At the end of the study, 8.3% of patients in Part A (who all received Xarelto) had a blood clot. In Part B, 1.6% of patients who received Xarelto had a blood clot compared to 8.8% of patients who received aspirin.

Xarelto was not studied and is not recommended for children younger than six months old who were born prematurely (birth before 37 weeks), have fed by mouth for less than 10 days, or weigh less than 5.7 pounds.

Safety Information

Xarelto must not be used in patients with active major bleeding or with serious allergic reactions to the medication. Early stoppage of any oral blood clotting treatment, including Xarelto, without switching to an alternative blood clotting treatment increases the risk of blood clots.

Xarelto can increase the risk of bleeding and can cause serious or fatal bleeding. Patients with some serious conditions or who take certain other drugs are at a higher risk of bleeding while using Xarelto.

Patients who receive neuraxial anesthesia (spinal/epidural anesthesia) or spinal puncture while taking medication to treat or prevent blood clots, such as Xarelto, are at risk of developing a hematoma (collection of blood by the spinal cord), which can result in long-term or permanent paralysis.

The most common side effects of Xarelto in children include bleeding, cough, vomiting and gastroenteritis (inflammation of the stomach and intestines).

See the prescribing information for additional information on risks associated with Xarelto.

Designations

Xarelto received priority review for this indication.