Two new guidance documents just released by the US Food and Drug Administration (FDA) finalize recommendations for Biologics License Applications (BLAs) and Investigational New Drug Applications (INDs) submitted in support of minimally manipulated placental or umbilical cord blood intended to help treat patients with hematopoietic system disorders.

Hematopoeietic system disorders include ones affecting red blood cells (e.g. anemia), granulocytes (e.g. granulocytopenia), lymphocytes (e.g. lymphocytosis), monocytes (e.g. monocytosis), platelets (e.g. thrombocytopenia) and 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 IND application to start a clinical trial or a BLA to initiate FDA's review of a tested product.

Draft versions of both guidance documents were released in June 2013, meant to replace an earlier 2009 guidance on the same topic. As Rafael Cassata, RAC wrote in Focus, the guidance documents included new indications for use of hematopoietic progenitor cells (HPC) derived from cord blood, such as hematopoietic and immunologic reconstitution in patients with hematopoeietic system disorders.

Those changes came about as the result of a 2011 meeting of FDA's Cellular, Tissue and Gene Therapies Advisory Committee, Cassata recalled.

Even so, the indications only cover HPCs if they were "minimally manipulated." Further manipulation means they can be regulated as drug products, per recent legal decisions.

The guidance also goes on to clarify various clinical data requirements for BLA and IND application submissions.

 

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 (FR)

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 (FR)