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BLA for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System: Guidance for Industry

FinalCenter for Biologics Evaluation and Research03/01/2014

Description

We, the Center for Biologics Evaluation and Research (CBER), FDA, are recommending ways that would allow you, the manufacturer, generally a cord blood bank, 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. This guidance document is intended to assist you in obtaining a biologics license. It contains information about the manufacture of minimally manipulated, unrelated allogeneic placental/umbilical cord blood and how to comply with applicable regulatory requirements.

Scope & Applicability

Product Classes

2
HPC, Cord Blood

Proper name for minimally manipulated, unrelated allogeneic placental/umbilical cord blood products.; Hematopoietic Progenitor Cells derived from placental/umbilical cord blood; Hematopoietic Progenitor Cells, Cord Blood product intended for reconstitution; The primary product subject to the guidance; Hematopoietic Progenitor Cells derived from umbilical cord blood; hematopoietic progenitor cells derived from cord blood; Hematopoietic Progenitor Cells from cord blood; hematopoietic progenitor ce

HCT/Ps

Human cells, tissues, or cellular or tissue-based products defined in 21 CFR Part 1271

Stakeholders

8
cord blood bank

The manufacturer typically responsible for applying for licensure.; Entity utilizing HLA testing laboratories

transplant centers

potential sponsors of IND applications

consignee

The receiving distributor who provides an acknowledgment letter.

laboratory director

responsible for all technical aspects of the cord blood bank

medical director

responsible for all medical aspects of the cord blood bank

Quality Control Unit

Responsible for ensuring the overall quality of the final drug product.

Contract Laboratory

Covered establishments include contract laboratories.; Entity performing testing only; Entities performing release or stability testing only

Manufacturer

Entity responsible for submitting NDINs

Regulatory Context

Attributes

6
minimally manipulated

Criterion for regulation under section 361

expiration date

Required label element for outsourcing facilities

expiration dates

Records of investigational products should include expiration dates if applicable.

Donor eligibility

ACTP availability for distribution only after donor eligibility is established

Dating Period

Should be 6 months from the date of collection.; Expiration or use-by period on container label

Potency

Measurement of potency for biological products

Identified Hazards

Hazards

4
Communicable Diseases

Risks that manufacturing controls are intended to prevent.

Microbiological contamination

Potential for contamination of covered produce; Known or reasonably foreseeable hazards in water

Contamination

Microorganisms or particulate matter that could adversely affect the device.

Cross-contamination

Risk associated with construction, traffic flow, and shared equipment.; Risk from raw food to RTE food or from insanitary objects; Personnel handling RTE foods touch contaminated surfaces

Related CFR Sections (20)

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See Also (8)