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FDA okays first-in-world valve to address congenital heart disease

Posted 26 March 2021 | By Kari Oakes 

FDA okays first-in-world valve to address congenital heart disease

The US Food and Drug Administration (FDA) announced Friday that it approved a first-in-world device to address pulmonary valve regurgitation in patients with some kinds of congenital heart disease, along with a device meant to help gait problems in people with multiple sclerosis.
The Harmony Transcatheter Pulmonary Valve (TPV) system is a less invasive option than open-heart repair for severe pulmonary valve regurgitation in patients with a native or surgically repaired right ventricular outflow tract (RVOT).
“The Harmony TPV provides a new treatment option for adult and pediatric patients with certain types of congenital heart disease. It offers a less-invasive treatment alternative to open-heart surgery to patients with a leaky native or surgically-repaired RVOT and may help patients improve their quality of life and return to their normal activities more quickly, thus fulfilling an unmet clinical need of many patients with congenital heart disease,” said Bram Zuckerman, MD, director of the Office of Cardiovascular Devices in FDA’s Center for Devices and Radiological Health (CDRH).
Severe pulmonary valve regurgitation can occur after open-heart procedures to repair congenital heart disease, which currently affects more than 2 million individuals in the US. Use of the TPV device may delay the need for further open-heart surgery and reduce the total number of surgeries needed over the lifetime for a person with congenital heart disease.
The device was approved via the premarket authorization pathway and was granted breakthrough device designation. In announcing its approval of the Harmony TPV device, FDA also noted that its development was part of the US-Japan Medical Device Harmonization by Doing Collaboration, “which seeks to promote timely access to innovative devices in both the U.S. and Japan through greater collaboration and communication among regulatory, academic and industry stakeholders across all stages of product development, and by developing practical solutions to shared real-world challenges.”
The Harmony TPV device is inserted, collapsed, via catheter into a major vein and then threaded to the RVOT, where deoxygenated blood exits the heart into the lungs via the pulmonary artery. When the valve is released from the catheter it self-expands and anchors into the RVOT, functioning like a native valve to prevent backflow of blood back into the heart.
The multi-center, prospective, non-randomized study FDA used for the basis of its premarket approval saw 70 patients receiving the TPV device. All patients met the primary safety endpoint of no procedure- or device-related death within 30 days of the implantation procedure. Of the patients who had echocardiograms that could be evaluated, 89.2% met the primary effectiveness endpoint of no additional device-related surgical or interventional procedures related to the device and “acceptable heart blood flow function” at the 6-month mark.
Almost one in four participants experienced arrhythmias, with 14.1% having ventricular tachycardia, a life-threatening rhythm disruption, after implantation. Leakage around the valve, minor bleeding, valve narrowing, and implant migration were less common adverse events. FDA's postmarketing requirements include following trial participants for 10 years.
Tongue stimulation for gait problems in MS
FDA also authorized a novel device that provides neural feedback to help with short-term improvement in the gait of patients with mild to moderate multiple sclerosis (MS). Helius Medical’s Portable Neuromodulation Stimulator (PoNS) is a prescription-only device meant to be used in conjunction with a therapeutic exercise program in patients 22 years old and up.
“Today’s authorization offers a valuable new aid in physical therapy and increases the value of additional therapies for those who live with MS on a daily basis,” said Christopher M. Loftus, MD, acting director of the Office of Neurological and Physical Medicine Devices in CDRH, in announcing the authorization.
The device rests on the tongue, held in place by teeth and lips, and uses electrical stimulation of the dorsal surface of the tongue to send information to the brain. The signals are sent based on data generated from a controller worn around the user’s neck that captures where the patient’s body is in space.
This neural feedback helps the user with balance and gait and may delay the need for other assistive technology for independent ambulation. Information about use of the device can also be uploaded to a computer and made available to therapists and others involved in the care of MS patients.
Helius Medical submitted its request for breakthrough device designation in August 2020. In October, FDA requested additional information, which Helius gathered and supplied.
In the end, the approval was based on two randomized, sham-controlled clinical trials involving a total of 44 patients with multiple sclerosis. The first study showed clinically and statistically significant improvement in a tool to measure gait deficits, while the second showed improvement in a measure of sensory organization tasks, but not gait.
FDA also considered a retrospective analysis of real-world data drawn from PoNS use by MS patients in clinical rehabilitation settings. This dataset added to the clinical trial data; FDA noted that “No serious safety adverse events were reported in the clinical studies or retrospective analysis of RWD.”
The safety recommendations for use of the PoNS devices are similar to those for other devices that use transcutaneous electrical nerve stimulation. FDA is imposing special controls including labeling and performance testing requirements for the PoNS device.
The PoNS device’s breakthrough device designation and approval came through the de novo premarket review pathway.
TPV announcement
PoNS announcement


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Tags: devices, FDA, US

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