May 16, 2019
On May 16, 2019, the Food and Drug Administration approved dalteparin sodium (FRAGMIN, Pfizer, Inc.) to reduce the recurrence of symptomatic venous thromboembolism (VTE) in pediatric patients 1 month of age and older.
This is the first FDA-approved therapy to treat VTE in pediatric patients.
Approval was based on evidence from two prospective, single-arm, multi-center clinical trials in 52 pediatric patients with or without cancer who had symptomatic deep vein thrombosis and/or pulmonary embolism. Patients were treated with dalteparin sodium subcutaneously twice daily for up to three months, with starting doses determined by age and weight. The starting dose of dalteparin sodium was based on population pharmacokinetic analysis of anti-Xa levels.
Efficacy was based on the achievement of therapeutic anti-Xa levels (0.5-1.0 IU/ml) and supported by the number of patients with lack of VTE progression or recurrent VTE. A total of 48 patients (92%) achieved a therapeutic anti-Xa level. Upon study completion, no patients experienced progression of the qualifying VTE and one patient (2%) had recurrence of VTE.
In pediatric patients with symptomatic VTE, the most common (greater than 10%) adverse reactions were injection site bruising (30%), contusion (12%), and epistaxis (10%).
Prescribing information contains a Boxed Warning alerting health care professionals and patients about the risk of epidural or spinal hematomas in patients who are anticoagulated with low molecular weight heparins or heparinoids and are receiving neuraxial anesthesia or undergoing spinal puncture.
The recommended dalteparin sodium dose in pediatric patients is based on age and weight. For details, view the full prescribing information for FRAGMIN.
FDA granted this application priority review. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by completing a form online at http://www.fda.gov/medwatch/report.htm, by faxing (1-800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088).