The Food and Drug Administration on Nov. 10 announced that Hemacord, a hematopoietic progenitor cells-cord preparation for allogenic hematopoietic stem cell transplantation, has received the first approval of a license for an umbilical cord blood therapy.
The approved indication is for "unrelated donor hematopoietic stem cell transplantation procedures ... in patients with disorders affecting the hematopoietic system." The FDA specified that the disorders could be inherited, acquired, or the result of myeloablative treatment, and that both adults and children could receive hematopoietic progenitor cells-cord (HPC-C) treatment.
Specific disorders eligible for HPC-C treatment include patients with hematologic malignancies, primary immunodeficiency diseases, bone marrow failure, and beta-thalassemia.
"Cord blood hematopoietic progenitor cell therapy offers potentially life-saving treatment options for patients with these types of disorders," said Dr. Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research, in a written statement.
The New York Blood Center produces Hemacord, and began providing HPC-C for clinical treatment in 1996 under an investigational new drug exemption from the FDA. As of early November, the center had supplied about 4,200 units of HPC-C for clinical treatments, said Christine Dingfelder, a center spokeswoman.
About a quarter of these stem-cell transplants involved an unrelated donor and recipient. HPC-C transplantation requires some degree of matching for human leukocyte antigens (HLA). By this fall, the center had collected and cryopreserved about 60,000 units of HPC-C that are available for future use, Ms. Dingfelder said.
The New York Blood Center currently collects human cord blood from consenting mothers at eight participating U.S. hospitals: six in the New York City area, one in Virginia, and one in Ohio. Each umbilical cord collection constitutes one unit. The center says its cord blood program is the world’s oldest and largest public cord blood bank.
Hemacord’s labeling will carry a boxed warning on the risks of graft-versus-host disease, engraftment syndrome, graft failure, and infusion reactions, each of which may be fatal. The labeling also specifies that patients who receive this product should be carefully monitored, and that a risk-benefit assessment, unit selection, and administration of Hemacord should be done under the direction of a physician who is experienced in hematopoietic stem cell transplantation.
Ms. Dingfelder is an employee of the New York Blood Center. Dr. Midthun did not have any disclosures.