US regulators approve first ever blood thinner for children to treat heart conditions and pediatric cancer
- On Thursday, the US Food and Drug Administration (FDA) approved Fragmin for kids over 1 month old
- The widely-used injection made by Pfizer prevents blood clots by targeting an enzyme that clots blood
- It was approved for adults in 1994, and doctors have been unofficially using it for kids
US regulators have approved the first blood-thinner for children to treat congenital heart disease, cancer, trauma and infection.
Until now, doctors have been unofficially using adult products to treat kids.
But the US Food and Drug Administration (FDA) has now approved the widely-used injection made by Pfizer, called Fragmin, to be used for pediatric patients over one month old.
‘Most children who have VTE are fighting a serious underlying primary illness such as cancer or congenital heart disease,’ director of the FDA’s Oncology Center of Excellence, Richard Pazdur, MD, said.
‘Not only are they fighting a serious illness, having a condition like VTE can then lead to significant complications and even death.’
After years of calls from doctors to provide an official treatment for kids, the FDA has given it a green light, following a three-month study on 38 pediatric patients that saw progress in 21, regression in seven, and no changes in two of the patients
The treatment, an infusion of dalteparin sodium, was approved for adults in 1994.
It blocks enzymes that fuel blood clots, lowering the risk of strokes and heart attacks.
Now, after years of calls from doctors to provide an official treatment for kids, the FDA has given it a green light, following a three-month study on 38 pediatric patients that saw progress in 21, regression in seven, and no changes in two of the patients.
Robert H. Pass, Chief of the Division of Pediatric Cardiology at the Icahn School of Medicine at Mount Sinai and Mount Sinai Kravis Children’s Hospital, hailed the move as ‘significant’.
‘At the present time, many and perhaps most medications that we use in pediatrics are being used in an “off label” manner,’ Dr Pass said.
That means that the drugs were ‘approved for adults but are being borrowed and utilized to treat children despite not being approved by the FDA for this purpose.’
Approving Fragmin for kids, he says, ‘represents one of the first agents used to treat venous thrombosis that has been formally approved for this indication in small children.’
‘We have used other unapproved agents for this purpose for over 40 years, particularly in very small children who have suffered a complication in the groin vessels following cardiac catheterization.
‘Having this agent approved for this use by the FDA is hopefully the first in many further approvals of agents for the treatment of venous thrombosis in children as well as other agents to treat many other pediatric diseases going forward.’