FDA approves 1st anticoagulant for use in pediatric patients

Fragmin (dalteparin sodium), a type of heparin that works as an anticoagulant, was approved by the FDA May 16 to treat symptomatic venous thromboembolism (VTE) in pediatric patients aged one month and up.

The FDA originally approved Fragmin, which is manufactured by Pfizer, in 1994, but that indication only applied to adults. The agency’s latest approval is for subcutaneous use of Fragmin in young patients with VTE, making the drug the first-ever blood thinner cleared for use in infants.

“Most children who have VTE are fighting a serious underlying primary illness such as cancer or congenital heart disease,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement. “Not only are they fighting a serious illness, having a condition like VTE can then lead to significant complications and even death. Prior to this approval, there had been no FDA-approved therapies to treat VTE in pediatric patients.”

Given that unmet need, Pazdur said Pfizer’s application for Fragmin was fast-tracked for priority review. The medication’s efficacy is based on a single trial of 38 pediatric patients with symptomatic deep vein thrombosis or pulmonary embolism, and after being treated with Fragmin for up to three months, 21 patients achieved resolution of their qualifying VTE, 7 showed regression, 2 showed no change and 1 experienced recurrence of VTE. No patients saw progression of their condition.

While Fragmin eases VTE, which is associated with increased risks of in-hospital mortality, recurrent VTE and post-thrombotic syndrome, it also comes with its fair share of side effects. Patients commonly experience bleeding, including hemorrhage, thrombocytopenia, hematoma or pain at the injection site, so the FDA advises they to undergo regular blood count lab tests and says healthcare providers should monitor their patients closely.