The Food and Drug Administration has licensed HEMACORD™ (Hematopoietic Progenitor Cells-Cord, HPC-C) for allogeneic hematopoietic (blood-forming) stem cell transplantation, the first such approval of a stem cell product in the world.
According to the Food and Drug Administration's notification to New York Blood Center (NYBC), HEMACORD™ is "indicated 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." Such conditions could include patients with hematologic malignancies, primary immunodeficiency diseases, bone marrow failure, and beta-thalassemia. HEMACORD™ is indicated for use with children.
"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 New York Blood Center (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. The mission of the NCBP is to facilitate access to cord blood as a treatment option for patients, notably those from diverse ethnic backgrounds.
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. 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 (collections, processing, testing, typing, cryopreservation, storage, distribution and shipping) cord blood units for clinical use. Further, it included the safety data from the transplantation of more than 4,000 cord blood grafts. In advance of the BLA submission, NCBP moved its entire manufacturing and storage resources to a state-of-the-art facility in Long Island City, New York.
"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. "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. Achieving systematic compliance with FDA Good Manufacturing Practices and Good Tissue Practices offers further assurance of the consistency, potency, purity, safety and efficacy of NCBP cord blood units."