FDA Approves First VTE Treatment for Pediatric Patients

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The U.S. Food and Drug Administration (FDA) gave Pfizer the green light to begin marketing its treatment for symptomatic venous thromboembolism (VTE) in pediatric patients. This is the first such medicine approved for this condition in pediatric patients.

On Thursday, the FDA approved Pfizer’s Fragmin (dalteparin sodium) injection for subcutaneous use to reduce the recurrence of VTE in patients one month of age or older. VTE can include a number of issues, such as deep vein thrombosis, which is a blood clot in the deep veins of the leg, as well as pulmonary embolism, a blood clot in the lungs, which can lead to death.

Fragmin, an anticoagulant, was initially approved by the FDA in 1994 for use with adults. The approval for pediatric patients, which was granted under priority review, was approved following a single trial with 38 pediatric patients with symptomatic deep vein thrombosis and/or pulmonary embolism. Following the completion of the study, 21 patients achieved resolution of the qualifying VTE, seven patients showed regression, two patients showed no change, no patients experienced progression of the VTE and one patient experienced recurrence of VTE, the FDA said in its announcement.

Symptomatic venous thromboembolism typically developed as a secondary complication from another medical issue, such as cancer, congenital heart disease, trauma and use of a venous catheter, among others. Pediatric VTE is associated with an increased risk of in-hospital mortality, recurrent VTE and post-thrombotic syndrome, or damage to the vein.

Richard Pazdur, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said that most children who have been diagnosed with VTE are fighting an underlying primary illness. Having a condition like VTE along with their primary medical concerns can lead to significant health complications or death, Pazdur said.

“Prior to this approval, there had been no FDA-approved therapies to treat VTE in pediatric patients. Given the unmet need, we granted the Fragmin application priority review and today we are approving it as the first anticoagulant (blood thinner) indicated for pediatric patients. We remain committed to advancing treatments for children with unmet medical needs,” Pazdur said in a statement.

Pfizer’s Fragmin does contain a boxed warning. The drug is associated with incidences of epidural or spinal hematomas, which is the accumulation of blood that can compress the spinal cord. This can occur in patients taking low molecular weight heparins (LMWH) or heparinoids and are receiving neuraxial anesthesia, or are undergoing spinal puncture. The spinal hematomas can result in long-term or permanent paralysis, the FDA said.

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