Draft Guidance Documents Cover FDA's Expectations for Blood Disorder Treatment Submissions

The US Food and Drug Administration (FDA) this week released two new guidance documents regarding the regulatory considerations for minimally manipulated, unrelated allogeneic placental and umbilical cord blood intended for patients with hematopoietic (blood) system disorders.

Hematopoeietic system disorders include ones affecting the:

• red blood cells (e.g. anemia)
• granulocytes (e.g. granulocytopenia)
• lymphocytes (e.g. lymphocytosis)
• monocytes (e.g. monocytosis)
• platelets (e.g. thrombocytopenia)
• clotting proteins (e.g. hemophilia)

The guidance documents are intended for sponsors of products intended to treat those conditions - assumed by FDA to be cord blood banks, registries, transplant centers, and physicians serving as sponsor-investigators - who are preparing to submit either an investigational new drug (IND) application to start a clinical trial or a biologics licensing application (BLA) to initiate FDA's review of a tested product.

Those products are what FDA calls "certain hematopoietic progenitor cells from placental/umbilical cord blood (HPC, Cord Blood)," and FDA has released several guidance documents over the year relating to their use.

The first draft guidance, which covers IND submissions, contains an overview of the required minimal information that must be submitted to FDA, including a description of the product's intended use, an explanation of why the HPC or cord blood units cannot be licensed, a description of the product, the clinical plan and donor eligibility information, and a summary of the product testing and manufacturing information.

The second draft guidance, which covers BLA submissions, is considerable more extensive, and covers all aspects of the product submission and what FDA says it is looking for.

Comments on both guidance documents are being accepted through 16 September 2013.

• Draft Guidance for Industry: Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and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
• Federal Register

• Draft Guidance for Industry and FDA Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for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
• Federal Register