Infant Life-Saving Drug Awaits FDA Approval

Omegaven, a medication developed at Boston Children’s Hospital to save the lives of infants who cannot absorb nutrition, is stuck in the FDA approval process, apparently causing delays that leave parents and doctors alike frustrated and worried.  The drug is a crucial part of treatment for Microvillus Inclusion Disease, a rare genetic condition in which a child cannot absorb fluid or nutrients except through a direct injection of a treatment called total parenteral nutrition, or TPN.  TPN, however, can cause liver damage over time; Omegaven counters that damaging effect.  More from NBC News:

The potentially life-saving medication Omegaven, an intravenous mixture made with fish oil, reduces the fatal fat accumulation in children’s livers caused by TPN. Fish oil contains anti-inflammatory omega-3 fatty acids, which have been shown to prevent fat buildup.

It is unclear when or whether Omegaven will be approved. The normal FDA process for approval is to test medications in large trials that randomly assign patients to receive either the new drug or a placebo. In fatal illnesses, that can present doctors with a tough ethical quandary: Do you do the science right and potentially lose some patients or just keep treating patients in research studies.

Not daunted by the lack of FDA approval, Sam [O’Connor's] family signed him up for a Boston Children’s Hospital research study looking at the new medication’s efficacy.

It didn’t take long to see results.

“For me, it was . . . the personality change,” Debra said. “To have him start responding to me and playing, it’s just like he’s actually a person again. You know, it’s almost like his life started at that point because before it was just enduring.”

Now 5, Sam is one of the lucky ones because he was able to get the drug he needed. Other children aren’t so lucky, says Puder, who developed the Omegaven treatment after watching up to four children die from liver failure each year at his hospital alone.

Without FDA approval, Omegaven is available only to those who can come to Boston to take the drug in a research protocol, or at another hospital with special dispensation from the FDA, a provision called “compassionate use.”

Image: Doctor holding infant, via Shutterstock

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