BLA for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood

Recommendation

9/10 May 2023
Heidelberg, Germany

Annex 2 & Co. - GMP Compliance for Biopharmaceuticals
Regulatory Requirements and Practical Implementation

This guidance for Industry - entitled Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System - should support manufacturers, generally cord blood banks licence procedure. It contents recommeded ways to apply for licensure of minimally manipulated, unrelated allogeneic placental/umbilical cord blood, for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment. The document should give assistance to get the biologics license and contents information about the manufacture of such cord blood and how to apply with the regulatory requirements.

This guidance finalizes the draft guidance of the same title dated June 2013 (June 17, 2013, 78 FR 36196) and supersedes the guidance entitled “Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications” dated October 2009 (October 20, 2009, 74 FR 53753) (2009 licensure guidance). The changes from the 2009 licensure 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, clarifying the types of clinical information that should be submitted for review, and adding additional references to other guidance documents on process validation and methods validation.

More details can be found directly in the guidance document.