Food and Drug Administration

Center for Biologics Evaluation and Research

Cellular, Tissue and Gene Therapies Advisory Committee

Meeting # 43

March 30, 2007

Guidance for Industry: Minimally Manipulated, Unrelated, Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution in Patients with Hematological Malignancies

Draft Advisory Committee Questions

1. Please discuss the types of data that could be submitted to demonstrate comparability between the previously manufactured HPC-C and HPC-C manufactured currently. The Draft Guidance states these data could include TNC count, viable CD34+ cell content, and colony forming unit content, other product attributes obtained from stability or other studies, data cited from the medical literature, and clinical outcome data. In the discussion please address the following:

• relationship of data obtained from viable CD34+ cell content and colony forming unit content
• alternate test methods
• the types of samples that are available from the previously manufactured HPC-C and HPC-C manufactured currently, and how they might impact comparability studies

2. Please comment on the clinical indication described in the Draft Guidance; i.e., hematopoietic reconstitution in patients with hematological malignances, and describe any additional data of which you are aware that could potentially support additional indications.

3. Please discuss any additional comments you have about the recommendations provided in the Draft Guidance to assist cord blood manufacturers in preparing information to be submitted in the Biologics License Application (BLA) for their HPC-C.

4. Please discuss what data would be adequate to demonstrate safety and efficacy of HPC-A, and to consider an approach to licensure of HPC-A similar to the one proposed for cord blood.