In an Oct. 1 news release, the U.S. Food and Drug Administration said it approved the Autus Size-Adjustable Valve, a surgically implanted pulmonary heart valve designed for pediatric patients with congenital pulmonary valve disease.

The device is the first heart valve designed to be expanded after implantation as a child grows, potentially reducing the need for repeated open-heart surgeries. It also is the first valve approved in the United States to use polymeric material, rather than animal-derived tissue, for its leaflets, the flaps that open and close to control blood flow.

“For far too long, children with congenital heart defects who need pulmonary valve replacement have had very limited surgical options, and those options have historically required multiple open-heart surgeries as children grow,” said Acting FDA Commissioner Kyle Diamantas, J.D. in the release. “Today’s landmark approval is a historic turning point and reflects the Trump Administration and the FDA’s commitment to help bring innovative, life-changing devices to the patients who need them most.”

Congenital heart defects affect about 1 in every 100 births in the United States. Pulmonary valve abnormalities include pulmonary valve stenosis, in which the valve is narrowed, and pulmonary valve atresia, in which the valve fails to form normally. Both conditions can restrict blood flow from the heart to the lungs. The Centers for Disease Control and Prevention estimates pulmonary valve atresia and stenosis together affect about 4,200 babies in the U.S. each year.

Treatment can improve blood flow and repair congenital defects, but some children develop pulmonary regurgitation, or leakage of blood back through the pulmonary valve. Over time, the condition can weaken or enlarge the right ventricle and lead to a need for pulmonary valve replacement as the child grows.

“For children born with congenital heart disease, valve replacement timing has always been a difficult decision — wait too long and you risk harm to the child, but intervene too early and they may need multiple open-heart surgeries as they grow,” said Michelle Tarver, M.D., Ph.D., director of the FDA’s Center for Devices and Radiological Health. “The Autus Size-Adjustable Valve gives doctors the option of far fewer trips to the operating room, which may result in better quality of life for these children and their families.”

The Autus valve can initially be implanted at approximately 13 millimeters in diameter, making it suitable for a toddler or preschool-aged child. As the child grows, doctors can expand the valve using a small inflatable tube, known as a balloon catheter. The valve can eventually reach 22 millimeters, comparable to an adult-sized pulmonary valve.

The valve’s polymeric leaflets also are intended to address a limitation of traditional valves, which typically use tissue derived from animals. That tissue can harden and break down more quickly in children, potentially leading to earlier repeat surgery. One-year follow-up data indicate the Autus valve’s synthetic leaflets have held up well over time. The FDA said it is the first time it has approved a valve using polymeric leaflet material for any indication.

The FDA based its approval on a clinical study involving 62 pediatric patients at 12 U.S. sites. At six months, the first 60 patients demonstrated acceptable blood flow through the right side of the heart and no more than mild leakage through the valve.

All patients in the study underwent a successful procedure. There were no deaths, blood clots requiring additional treatment, strokes or other clot-related complications. Three patients experienced a fracture in the valve frame and two experienced reduced movement of a valve leaflet, but none of those events caused symptoms.

Valve performance began to deteriorate in two patients who started to outgrow their valves. Both valves were successfully expanded without an invasive surgical procedure, although the FDA said experience with the approach remains limited. Longer-term studies are underway to better understand the risks and determine whether valve expansion can maintain a functional valve while reducing the need for additional surgeries.

The Autus Size-Adjustable Valve is the first pediatric approval to come through the FDA’s Total Product Life Cycle Advisory Program, a voluntary program designed to encourage faster development of high-quality, safe, effective and innovative medical devices. The FDA previously granted the device Breakthrough Device designation, which is intended to expedite the development and review of devices that may provide more effective treatment or diagnosis for life-threatening or irreversibly debilitating conditions.

The valve was approved through the FDA’s premarket approval pathway, the agency’s most stringent medical device marketing application process. The pathway applies to certain Class III devices, which generally pose the greatest risk. The FDA reviews scientific evidence, including nonclinical and clinical data, to determine whether there is reasonable assurance a device is safe and effective for its intended use and whether its benefits outweigh known risks.

Edwards Lifesciences received approval for the Autus Size-Adjustable Valve, which was developed by Autus Valve Technologies Inc.