Regulatory Affairs Professionals Society – The US Food and Drug Administration (FDA) on Monday offered one of its first Regenerative Medicine Advanced Therapy (RMAT) designations to Humacyte’s investigational human acellular vessel (HAV) Humacyl.
“Being one of the first companies to receive the Regenerative Medicine Advanced Therapy Designation from the FDA is an honor and a testament to the significant potential for Humacyl to address a great unmet medical need for patients who are undergoing dialysis,” Carrie Cox, chairman and CEO of Humacyte, said in a statement.
Cox also told Focus: "The designation provides Humacyte with additional resources from the FDA to expedite the review pathway, similar to actions that expedite the development and review of a marketing application for drugs under the Breakthrough Therapy designation...Additional support includes timely advice and interactive communications with FDA, as well as proactive and collaborative involvement by senior FDA managers and experienced review and regulatory health project management staff.
To obtain such a designation, the treatment must be “a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products,” intended to “treat, modify, reverse, or cure a serious or life-threatening disease” and have preliminary clinical evidence that the drug has the potential to address unmet needs.
The company says it was granted the RMAT designation, which is the first publicly-disclosed RMAT designation granted, because Humacyl meets these criteria for the creation of vascular access in performing hemodialysis.
A Phase III trial of Humacyl is ongoing for an expanded polytetrafluoroethylene (ePTFE) graft in 350 patients with kidney failure who are not candidates for fistula placement. The trial involves approximately 35 sites in the US, EU and Israel. The Phase III study’s primary objective is to compare the durable use (secondary patency) of the HAV with that of the ePTFE graft during its use as a conduit for hemodialysis, Humacyte said.
"Strong early clinical data from Humacyl Phase II studies supported our designation. The team’s work in progressing the clinical proof-of-concept for Humacyl in hemodialysis access required patience, research, diligence, and innovation," Cox added.