FDA News

Cell therapy receives orphan drug designation for chronic heart failure

The FDA granted orphan drug designation to rexlemestrocel-L for the prevention of postimplantation mucosal bleeding in patients with end-stage chronic heart failure who require a left ventricular assist device.

Rexlemestrocel-L (Revascor; Mesoblast) is an investigational allogeneic mesenchymal precursor cell therapy under development for injection into the heart muscle in patients with moderate to advanced or end-stage chronic HF.

“We are very pleased that the FDA has granted our heart failure cell therapy product candidate, Revascor, orphan drug designation for use in patients with LVADs to prevent mucosal bleeding, including from the gastrointestinal tract,” Silviu Itescu, MBBS, FRACP, chief executive of Mesoblast, said in a press release. “We look forward to our upcoming meeting with the FDA to discuss a potential approval pathway under the product’s existing Regenerative Medicine Advanced Therapy (RMAT) designation for this life-threatening condition.”

The FDA granted RMAT designation to rexlemestrocel-L in December 2017, based on the results of a pilot study of 30 patients with end-stage HF who required a left ventricular assist device, according to a company statement. The study, conducted by the NIH, showed that a single intramyocardial dose of rexlemestrocel-L reduced GI bleeding and associated hospitalizations by 70%.

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 Ltd. Mesoblast heart failure cell therapy receives ophran drug designation from FDA for prevention of gastrointestinal bleeding in patients with left ventricular assist devices. Available at: investorsmedia.mesoblast.com/static-files/c08cebeb-456b-49d4-8e0d-cfc5df110d9e. Accessed June 24, 2019.

The FDA granted orphan drug designation to rexlemestrocel-L for the prevention of postimplantation mucosal bleeding in patients with end-stage chronic heart failure who require a left ventricular assist device.

Rexlemestrocel-L (Revascor; Mesoblast) is an investigational allogeneic mesenchymal precursor cell therapy under development for injection into the heart muscle in patients with moderate to advanced or end-stage chronic HF.

“We are very pleased that the FDA has granted our heart failure cell therapy product candidate, Revascor, orphan drug designation for use in patients with LVADs to prevent mucosal bleeding, including from the gastrointestinal tract,” Silviu Itescu, MBBS, FRACP, chief executive of Mesoblast, said in a press release. “We look forward to our upcoming meeting with the FDA to discuss a potential approval pathway under the product’s existing Regenerative Medicine Advanced Therapy (RMAT) designation for this life-threatening condition.”

The FDA granted RMAT designation to rexlemestrocel-L in December 2017, based on the results of a pilot study of 30 patients with end-stage HF who required a left ventricular assist device, according to a company statement. The study, conducted by the NIH, showed that a single intramyocardial dose of rexlemestrocel-L reduced GI bleeding and associated hospitalizations by 70%.

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 Ltd. Mesoblast heart failure cell therapy receives ophran drug designation from FDA for prevention of gastrointestinal bleeding in patients with left ventricular assist devices. Available at: investorsmedia.mesoblast.com/static-files/c08cebeb-456b-49d4-8e0d-cfc5df110d9e. Accessed June 24, 2019.