13/14 December 2022
On 6 March, the FDA published a new Guidance for Industry and FDA Staff - Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System.
This document is supposed to give advice for cord blood banks, registries, transplant centers or other sponsors for the submission of an Investigational New Drug Application (IND). It is especially applicable if these placental/umbilical cord blood (HPC, Cord Blood) units are not licensed in accordance with Title 21 of the Code of Federal Regulations Part 601 (21 CFR Part 601). It fits if a suitable human leukocyte antigen (HLA) matched cord blood transplant is needed for hematopoietic and immunologic reconstitution in patient. That means patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment and if there is no satisfactory alternative treatment available.
This guidance finalizes the draft guidance of the same title dated June 2013, (78 FR 36194) and supersedes the guidance entitled “Guidance for Industry and FDA Staff: Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications” dated June 2011 (August 2, 2011, 76 FR 46303) (2011 IND guidance). The changes from the 2011 IND guidance include replacing the term “HPC-C” with the term “HPC, Cord Blood,” which is the proper name for these products, expanding the clinical indication, and clarifying the applicability of the requirement for a table of contents in the IND submission.