November 11, 2011
Cord Blood Treatment Receives FDA Approval
US drug regulators for the first time have approved a therapy using cells of human blood from the placenta and the umbilical cord to treat cancer or blood-forming disorders, reports ABC News.
The US Food and Drug Administration (FDA) on Thursday officially licensed HEMACORD (Hematopoietic Progenitor Cells-Cord, HPC-C) for “use in unrelated donor hematopoietic progenitor cell transplantation procedures“¦ in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment,” according to a notification to the NY Blood Center.
Last year a study was published stating that cord blood was comparable to bone marrow or peripheral blood progenitor cells in treating acute leukemia in adults, with similar effects on leukemia-free survival.
The progenitor cells are infused into patients and make their way to bone marrow, where they divide and mature. As they move into the bloodstream, they can help build new blood cells or restore their capacities, including immune function.
The FDA implemented a two-year phase-in period in 2009 for manufacturers to submit either a license application or new drug application, which ended on October 20, 2011.
HEMACORD´s approval was based on safety and effectiveness data submitted by the company.
HEMACORD is approved for use in blood-restoring stem cell transplants, which can use cells from three sources: cords, bone marrow and peripheral blood, the flowing blood that circulates through the body.
The product will carry a boxed warning about the risks of graft-versus-host disease, engraftment syndrome, graft failure, and infusion reactions, each of which may be fatal, the FDA warned.
Patients who receive HEMACORD should be monitored, with both risk and benefit assessment and administration performed “under the direction of a physician experienced in hematopoietic stem cell transplantation,” the FDA noted.
“We have been using cord blood for years,” said Dr. Machi Scaradavou, medical director of the NYBC's National Cord Blood Program. “Recently, FDA decided that it needs to be licensed and this is the first cord blood product and stem cell product to be licensed.”
“We are thrilled to be the first public cord blood bank with an FDA-licensed product for transplantation,” said Christopher D. Hillyer, MD, President and CEO of NYBC.
Since 1996, NYBC´s National Cord Blood Program (NCBP) at the Howard P. Milstein National Cord Blood Center has provided cord blood units for transplantation under an Investigational New Drug (IND) exemption from the FDA.
To obtain FDA licensure of HEMACORD all collection and manufacturing conditions of the cord blood units met FDA standards for product potency, purity and efficacy, and patient safety.
“The application process has been a rigorous and educational one for all of us at the National Cord Blood Program,” said Pablo Rubinstein, MD, NYBC Vice President and Program Director of NCBP.
NCBP´s Biologics License Application (BLA) included extensive documentation of the systems and controls used, and the validations performed to ensure compliance with FDA standards in all steps of manufacturing. Further, it included the safety data from the transplantation of more than 4,000 cord blood grafts.
“We have always understood that providing high quality cord blood units for clinical use requires thorough scientific and technical definitions of methods, detailed analysis of results, and hard work,” Rubinstein added.
In advance of the BLA submission, NCBP moved its entire manufacturing and storage resources to a new facility in Long Island City, New York.
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