Neuromodulator and artificial kidney to be reviewed under FDA's Expedited Access Pathway

The Wearable Artificial Kidney (WAK)--Courtesy of Stephen Brashear

A neuromodulator made by Boston's PathMaker Neurosystems and Blood Purification Technologies' Wearable Artificial Kidney just became some of the first devices to be accepted into the FDA's Expedited Access Pathway for "breakthrough" devices. The development is a win for both companies, and also for the FDA, whose efforts to promote the 6-month-old pathway have resulted in only one other device making it into the EAP pipeline.

The PM-2200 neuromodulator is being developed to treat muscle spasticity in patients with neurological conditions like stroke, cerebral palsy, multiple scelorosis and spinal cord injury, among others.

Meanwhile, the Wearable Artificial Kidney is intended to help the 600,000 U.S. patients with end-stage renal disease, which has a mortality rate of 20%. The device was invented by nephrologist Dr. Victor Gura, of Beverly Hills, CA.

Entrance into the program should mean a faster approval (or possible rejection) because the FDA says on the EAP website that it "may accept a greater degree of uncertainty of the benefit-risk profile for these devices if the uncertainty is sufficiently balanced by other factors, including the probable benefits for patients to have earlier access to EAP Devices, and adequate postmarket controls to support premarket approval."

The expedited pathway is for devices intended "to treat or diagnose a life-threatening of irreversibly debilitating disease or conditions" that offer a clinically meaningful advantage over approved alternatives. In addition, for an investigational device to earn EAP status, the FDA must approve its data development plan.

"Through the FDA Expedited Access Pathway program, we will be able to more rapidly bring to market this breakthrough technology for treating muscle tone disorders," said PathMaker CEO Dr. Nader Yaghoubi in a statement. "As one of the first companies selected into this program, we look forward to working with FDA to rapidly make MyoRegulator available to patients and clinical institutions worldwide."

Gura says the externally worn Wearable Artificial Kidney will reduce hospitalizations for end-stage renal disease patients, who currently need to spend up to 12 hours per week strapped to a kidney dialysis machine. Human trials of the device have been conducted at the University of Washington at Seattle, and details will be presented this week at the American Society of Nephrology's annual conference in San Diego.

"We are thrilled that the FDA has awarded the Wearable Artificial Kidney with its new Expedited Access Pathway," said Gura in a statement. "The WAK has been in development for 14 years, and this designation brings us one vital step closer to making it available to the public. Soon, we hope to be improving the lives of hemodialysis patients in the U.S. and around the world with the WAK."

The development should please the FDA just as much as it does Yaghoubi and Gura. The head of the agency's device arm (CDRH), Dr. Jeff Shuren, regularly promotes the EAP program as a critical plank in of his plan to promote faster approval times. Benefits of the program include priority access to FDA reviewers and the possibility for shorter clinical trials via the use of intermediate or surrogate endpoints.

But industry lawyers have told FierceMedicalDevices they have some reservations about the program. Devices approved through the EAP program are more likely to receive tough postmarket surveillance, but the final guidance, issued in April 2015, does not specify what would happen if the postmarket data is troubling or disappointing.

At the annual AdvaMed industry conference, Shuren told FierceMedicalDevices that in such a scenario, the FDA would potentially withdraw the device's approval, just as it would if the product were approved under conventional pathways.

AdvaMed has given EAP program a lukewarm reception. Following the issuance of the draft guidance in April 2014, AdvaMed said the pathway does not meet Congress intent, as spelled out under the FDA Modernization Act of 1997.

The trade association successfully lobbied for the inclusion of a more ambitious accelerated device pathway in the 21st Century Cures Act to overhaul drug and device industry regulation; the comprehensive bill easily passed the House, but has yet to come up for a vote in the Senate.

Another industry concern is the possibility for an overly narrow indication as a result of the shorter trial.

Still, there are now three known devices being reviewed under the EAP initiative as it approaches its half-year anniversary. In June, Cohera Medical announced that its Sylys resorbable synthetic sealant to prevent leakages during gastrointestinal surgical procedures is being reviewing under the pathway.

Industry watchers should keep a close on the MyoRegulator neuromodulator, Wearable Artificial Kidney and Sylys surgical sealant, as the EAP program is emblematic of the broader approach that Shuren wants to take as head of the FDA's device arm. The model (already being employed in Europe) is characterized by faster premarket decision-making balanced by increased postmarket surveillance.

- read the release from Blood Purification Technologies
- read the the release from PathMaker Neurosystems

Editor's Note: Upon the issuance of a new and corrected press release, this article was changed to reflect the sponsor of the Wearable Artificial Kidney. It is Blood Purification Technologies, not Cedars-Sinai Medical Center.