EMA Releases New Guidance on Stem Cell Products

On Feb. 4, 2011, the European Medicines Agency released a new document on drug products that are manufactured using stem cells. Titled, Reflection Paper on Stem Cell-Based Medicinal Products, the document advises manufacturers on quality-control issues regarding these products and provides an overview of the use of stem cells in drug development. The final document is based on a draft paper issued in March 2010.

The paper applies directly to those companies pursuing marketing authorization for stem cell-based products. According to the document, the paper “should be read in conjunction with existing guidance on cell-based medicinal products (Guideline on human cell-based medicinal products [EMEA/CHMP/410869/2006]) which addresses general aspects of cell-based medicinal products.”

Specifically, the paper provides key quality considerations for manufacturers, including purity of the stem cells. The document states that, “the aim should be to maximize the active components and minimize features which do not contribute, or may negatively impact on therapeutic activity/safety.” For example, the document points out that tumor formation is an inherent risk when using pluripotent stem cells. This possibility should be assessed during product development and the presence of pluripotent cells should be limited and justified, states the document.  The guidance also addresses cell rejection, including using potential immune responses of drug product to assess the risk of rejection and elimination of the stem cell.

EMA’s Committee for Advanced Therapies adopted the final reflection paper on Jan. 14, 2011. Comments from a May 2010 public workshop and a public consultation period were considered in drafting the final document.