The US Food and Drug Administration (FDA) has granted Humacyte’s Humacyl investigational human acellular vessel (HAV) Regenerative Medicine Advanced Therapy (RMAT) designation.
This designation means that the FDA will help facilitate the efficient development and expedited review of the HAV for vascular access to patients in need of life-sustaining haemodialysis. Carved from the model of other FDA expedited programs such as Breakthrough Therapy, the RMAT designation is a new expedited program for regenerative medicine products.
According to a company release, since receiving the FDA’s Fast Track Designation in 2014, Humacyte has continued to develop clinical evidence indicating that it has the potential to address unmet medical needs for haemodialysis patients by decreasing vascular access complications, that in turn may result in more favourable morbidity and mortality outcomes.
Humacyte is currently undertaking a Phase III clinical trial of Humacyl compared to 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, Europe 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 haemodialysis. The company published Phase II results in May 2016, showing that Humacyl may have the potential for long-term use and safety in patients suffering from ESRD who require renal replacement therapy and are not candidates for fistula.
The RMAT designation provides for additional resources from the FDA, in an effort to expedite the review pathway. The designation makes a product eligible for the same actions to expedite the development and review of a marketing application that are available to drugs that receive breakthrough therapy designation, including 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. A product designated as an RMAT also may be eligible for other FDA expedited programs, such as Priority Review. FDA also may conduct a rolling review of products in its expedited programs, reviewing portions of a marketing application before the complete application is submitted.
The FDA’s RMAT designation was a primary component of the 21st Century Cures Act, signed into law by US president Barack Obama in December 2016 and championed by former vice president Joe Biden. According to this legislation, eligible drugs include regenerative medicine therapy, further defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or a combination product using such therapies or products. Further, eligible drugs are those intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and present preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such disease or condition. Humacyte was granted the RMAT designation through Humacyl meeting these criteria for the creation of vascular access in performing haemodialysis.
Humacyte is also developing and seeking regulatory approval for additional HAV applications, and recently announced the commencement of a US Phase II clinical trial of the bioengineered vessel as a bypass graft in patients with peripheral arterial disease.