Berlin Heart GmbH, a closely held German device maker, said it won U.S. regulatory approval for the first heart pump used to help children survive as they await a transplant.
The product, known as a ventricular assist device, does the work of a normal heart in children with congenital or other cardiovascular defects that prevent their own from circulating enough blood to fuel the body. The device, named Excor, is already approved in Europe and Canada, the company said today in a statement.
The system serves as bridge to a transplant, keeping the children alive and their organs functioning while they wait for a donor heart. While adults can receive similar products sold by Framingham, Massachusetts-based HeartWare International Inc. and Pleasanton, California-based Thoratec Corp., the device known as the Berlin Heart is the only one approved for children.
“This is a landmark event for children suffering from terminal heart failure,” said Charles Fraser Jr., head of congenital heart surgery at Texas Children’s Hospital and professor of surgery and pediatrics at Baylor College of Medicine in Houston. “The medical community is now able to offer this lifesaving device to support desperate children who would not otherwise survive while awaiting a heart transplant.”
The Berlin Heart was tested in 48 children across the U.S. starting in November 2007, the first-ever pediatric trial of a mechanical heart pump. A Food and Drug Administration advisory panel recommended approval in July.
Few Treatment Options
Children with heart failure have few other treatment options. While an instrument similar to a heart-lung bypass machine can work for a few weeks, the children must be sedated. The Berlin Heart enables them to be alert and active.
FDA approval allows patients immediate access to the heart pump, rather than requiring them to apply for compassionate use of the device that can take a week or longer.
“Having to wait to get compassionate care approval can be the difference between whether a kid lives or dies,” said Traci Reichman, the mother of 9-year-old Lane Eberhardt, who received a Berlin Heart on May 3, 2010. “If it’s readily available, it makes a huge difference.”
Lane was born with critical aortic stenosis, a narrowing of the key artery that obstructed his blood flow. He underwent numerous treatments to expand, repair and replace the valve, without success, Reichman said in a telephone interview.
Lane was admitted to the hospital at the Cleveland Clinic on Feb. 4, 2010, and given medication to help his heart function while he waited for a transplant. By late April, he weighed 35 pounds and doctors said he had only days to live if they didn’t intervene. It took a week to get FDA approval for compassionate use of the Berlin Heart, and his doctor sat beside his bed the night before his implant to make sure he survived to surgery.
“He laid in a fetal position most of the time prior to the machine being attached to his heart because he was in so much pain,” she said. “He became a normal kid with a small attachment. For eight months, that machine did everything for him, until we got a call that we had a donor.”
He underwent a heart transplant on Dec. 22, almost a year ago. Now Lane is up to 75 pounds, back in school and playing baseball, she said. This year, he’s trying wrestling.
More than 800 kids ranging in age from newborns to teenagers worldwide have received the heart pump implants, according to company documents. While the device is supposed to work as a bridge to transplant, some children have recovered enough after receiving a Berlin Heart that they are able to have the pump removed and their own heart takes over the workload.
Until now, the device could be implanted only at fewer than a dozen children’s hospitals in the country that participated in its clinical trials, said Robert Stewart, chair of pediatric thoracic and cardiovascular surgery at the Cleveland Clinic’s Children’s Hospital. Doctors had to call the company and write the FDA letters for permission before the approval, he said in a telephone interview.
“There were individuals who could benefit but didn’t get it because of the hurdles,” said Stewart, estimating that 150 to 200 children are waiting for treatment. “These are relatively small numbers compared to the thousands of adult cases in the United States, but it will mean a huge difference for those individuals.”