An FDA advisory committee has recommended in favor of a humanitarian exemption for Berlin Heart's EXCOR ventricular assist device for use in children as a bridge to heart transplant.
Panel members voted unanimously, 16-0, that the device is safe for use in patients who meet eligibility criteria, and also voted unanimously that the device's benefits outweigh its risks.
The device is intended for use by pediatric patients with severe isolated left ventricular or biventricular dysfunction who are candidates for cardiac transplant and who require circulatory support. The panel compared survival on the device with treatment with extracorporeal membrane oxygenation (ECMO).
The FDA briefing documents discussed results of a study of 48 children awaiting heart transplants; the children were divided into two cohorts depending on their age.
In the first cohort, which included children ages 30 days to 4 years, 87.5% of the children put on EXCOR according to the study protocol survived to transplant or successful weaning, compared with 75% of those receiving ECMO (HR [unadjusted for matching] was 0.043,P=0.004).
In the second cohort, which included children ages 4 to 16, 91.7% of those on the EXCOR device survived to transplant or weaning, compared with 66.7% of those on ECMO (HR [unadjusted for matching] 0.02, P=0.004).
In making its decision, the panel pointed to observational data showing that patients could remain on the EXCOR device for longer periods of time compared to ECMO, according to a summary of the meeting provided by the FDA.
The panel also was persuaded by the fact that the EXCOR patients experienced fewer adverse events than the ECMO patients, according to the summary.
There was concern expressed, however, that the EXCOR patients experienced a higher percentage of acute strokes and adverse neurological outcomes compared with the ECMO patients, and the panel recommended that a post-approval study of that aspect of the results be conducted.
The panel also recommended that pediatric patients receiving the EXCOR device be enrolled in registries that would collect data for about five years on stroke, pump thrombus, and longer-term neurologic and quality of life outcomes.
American Heart Association spokesperson Mariell Jessup, MD, who is a professor of medicine at the University of Pennsylvania Heart and Vascular Center hailed the advisory panel's action and said that if the FDA follows the recommendations of the advisory committee it would provide "ventricular assist devices manufactured to fit the smallest of children with end-stage heart failure-allowing their physicians to sustain their fragile lives while donor hearts can be found. This is a strategy that has been available for years for adult and larger children, and now can be offered for a wider group of young patients."
The American College of Cardiology also expressed its support for the EXCOR device. "While the literature documents potential risks to the implantation of ventricular assist devices in the pediatric population, it also indicates that ventricular assist device implantation offers the opportunity to extend the time limits of therapy beyond ECMO and maximal medical therapy as children wait for a new heart to become available," David R. Holmes Jr., MD, president of the ACC, wrote in a letter to FDA Commissioner Margaret Hamburg, MD.
"Adult VADs are simply not appropriate for the pediatric population, and there are no other devices that can be used as a substitute. We hope the FDA will encourage Berlin Heart and other device manufacturers to develop additional pediatric cardiology devices that appropriately treat the needs of this special population."
The FDA does not have to follow the advice of its advisory committees, but often does.
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