The long-awaited "Berlin Heart," a ventricular assist device for infants and children with heart failure, has been approved in the United States.
The mechanical pulsatile cardiac assist device, which comes in different sizes to fit children from newborns to teenagers, was approved by the Food and Drug Administration on Dec. 16, the agency announced in a statement.
"This is a step forward, it is the first FDA-approved pulsatile mechanical circulatory support device specifically designed for children," Dr. Susan Cummins, chief pediatric medical officer in the FDA’s Center for Devices and Radiological Health, said in the statement. "Previous adult heart assist devices were too large to be used in critically ill children to keep them alive while they wait to get a new heart."
The device, commonly referred to as the Berlin Heart, is the EXCOR Pediatric System, and is manufactured by Berlin Heart, a German company. The device consists of one or two external pneumatic blood pumps, tubes to connect these pumps to the chambers of the heart and the great arteries, and a driving unit, according to the FDA.
Use of the EXCOR device improved survival to transplant among patients in a U.S. study of 48 pediatric patients, compared with those treated with the current standard of care, extracorporeal membrane oxygenation (ECMO), the agency statement said. Stroke is a risk associated with use of the EXCOR device.
It was approved under a Humanitarian Device Exemption, which requires proof that that "the probable benefit from use of the device outweighs the probable risk of illness or injury from its use to obtain the FDA’s approval," the agency said. HDE-approved devices are subjected to certain use restrictions. (Standard approval of devices requires that "reasonable assurance of effectiveness" be shown).
The FDA’s Office of Orphan Products Development designated this product as a Humanitarian Use Device (HUD) because it is intended to benefit patients with a disease or condition that affects fewer than 4,000 people in the United States per year, the definition of an orphan product.
One of the study sites was Texas Children’s Hospital, Houston. In a statement release by the hospital, Dr. Charles D. Fraser Jr., the hospital’s surgeon in chief, said that the approval "ushers in a new era for children with terminal heart failure. The medical community is now able to offer this life-saving device to support desperate children who would not otherwise survive while awaiting donor hearts."
Because of the small number of pediatric-size donor hearts available, the median wait time for a donor heart for infants is 119 days; 12%-17% of children and 23% of infants on heart transplant lists die before a heart becomes available, according to the FDA.