World-first approval covers acquired and inherited hematopoietic system disorders & those resulting from myeloablative therapy.
FDA approved the New York Blood Center’s (NYBC) human cord blood-derived hematopoietic progenitor cell (HPC) product, Hemacord™, for use in allogeneic hematopoietic stem cell transplantation. The approval, which NYBC says marks the first for a stem cell product globally, means Hemacord cells can be used for unrelated hematopoietic progenitor cell transplantation procedures in patients with inherited and acquired disorders affecting the hematopoietic system, and those resulting from myeloablative therapy. Potential patient sets could include those with blood cancers, primary immuhnodeficiency diseases, bone marrow failure, and beta-thalassemia.
NYBC’s BLA for Hemacord included documentation of the systems and controls used and validations to ensure compliance with FDA standards at all steps of manufacturing cord blood units for clinical use, combined with safety data from the transplantation of over 4,000 cord blood grafts. Prior to submitting the BLA, the NYBC’s National Cord Blood Program (NCBP) transferred its entire manufacturing and storage resources to a facility in Long Island City, NY.
“The application process has been a rigorous and educational one for all of us at the NCBP,” comments Pablo Rubinstein, M.D., NYBC’s vp and program director at the NCB. “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.”
FDA issued a guidance document in 2009 to help manufacturers applying for licensure of certain cord blood units and instituted a two-year phase-in period for manufacturers of cord-blood derived hematopoietic progenitor cells to either submit a license application or an IND application. The phase-in period ended on October 20.