FDA News

FDA provides pathway to BLA approval for chronic heart failure cell therapy

The FDA has established a pathway to marketing for Revascor, an investigational cell therapy used for the prevention of postimplantation mucosal bleeding in patients with end-stage chronic heart failure who require a left ventricular assist device.

Revascor (rexlemestrocel-L, Mesoblast) is an allogeneic mesenchymal precursor cell therapy for injection into the heart muscle in patients with moderate to advanced or end-stage chronic HF. Mesoblast reported “positive outcomes” from a recent meeting with the FDA to support product authorization via a biologics license application, according to a company press release.

Mesoblast said the FDA confirmed that the company’s recently completed placebo-controlled trial of rexlemestrocel-L achieved the primary end point of reducing major mucosal bleeding events and supported its BLA. The FDA also agreed to the establishment of a confirmatory phase 3 clinical trial of rexlemestrocel-L in patients with LVAD that will include a primary endpoint of major mucosal bleeding and secondary endpoints evaluating cardiovascular function.

The phase 3 trial will be conducted with the International Center for Health Outcomes Innovation Research (InCHOIR) at the Icahn School of Medicine at Mount Sinai in New York, according to Mesoblast.

“The FDA guidance on the pathway to registration for our heart failure product candidate in LVAD patients is a major step forward for our cardiovascular program,” Silviu Itescu, MBBS, FRACP, chief executive of Mesoblast, said in a press release.

“We will work closely with the FDA and InCHOIR to generate the confirmatory clinical data needed for full marketing approval of [rexlemestrocel-L] in the prevention of this life-threatening inflammatory complication of an LVAD implant in end-stage heart failure patients,” he added.

Rexlemestrocel-L has previously received both FDA regenerative medicine advanced therapy (RMAT) and orphan drug designations.

The FDA’s RMAT designation program is part of the 21st Century Cures Act and was created to expedite regenerative medicine therapies — defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations — that is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition. The therapy must have preliminary clinical evidence indicating the drug has the potential to address unmet medical needs for such a disease or condition.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

Reference:

Mesoblast. FDA provides guidance on clinical pathway to marketing application for Revascor in end-stage heart failure patients with an LVAD [press release]. August 27, 2019.  Available at: http://investorsmedia.mesoblast.com/static-files/2b541ce3-2902-4dff-9228-61895019f534. Accessed August 28, 2019.

The FDA has established a pathway to marketing for Revascor, an investigational cell therapy used for the prevention of postimplantation mucosal bleeding in patients with end-stage chronic heart failure who require a left ventricular assist device.

Revascor (rexlemestrocel-L, Mesoblast) is an allogeneic mesenchymal precursor cell therapy for injection into the heart muscle in patients with moderate to advanced or end-stage chronic HF. Mesoblast reported “positive outcomes” from a recent meeting with the FDA to support product authorization via a biologics license application, according to a company press release.

Mesoblast said the FDA confirmed that the company’s recently completed placebo-controlled trial of rexlemestrocel-L achieved the primary end point of reducing major mucosal bleeding events and supported its BLA. The FDA also agreed to the establishment of a confirmatory phase 3 clinical trial of rexlemestrocel-L in patients with LVAD that will include a primary endpoint of major mucosal bleeding and secondary endpoints evaluating cardiovascular function.

The phase 3 trial will be conducted with the International Center for Health Outcomes Innovation Research (InCHOIR) at the Icahn School of Medicine at Mount Sinai in New York, according to Mesoblast.

“The FDA guidance on the pathway to registration for our heart failure product candidate in LVAD patients is a major step forward for our cardiovascular program,” Silviu Itescu, MBBS, FRACP, chief executive of Mesoblast, said in a press release.

“We will work closely with the FDA and InCHOIR to generate the confirmatory clinical data needed for full marketing approval of [rexlemestrocel-L] in the prevention of this life-threatening inflammatory complication of an LVAD implant in end-stage heart failure patients,” he added.

Rexlemestrocel-L has previously received both FDA regenerative medicine advanced therapy (RMAT) and orphan drug designations.

The FDA’s RMAT designation program is part of the 21st Century Cures Act and was created to expedite regenerative medicine therapies — defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations — that is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition. The therapy must have preliminary clinical evidence indicating the drug has the potential to address unmet medical needs for such a disease or condition.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

Reference:

Mesoblast. FDA provides guidance on clinical pathway to marketing application for Revascor in end-stage heart failure patients with an LVAD [press release]. August 27, 2019.  Available at: http://investorsmedia.mesoblast.com/static-files/2b541ce3-2902-4dff-9228-61895019f534. Accessed August 28, 2019.