Stem-cell Breakthrough in the EU

The first veterinary stem cell products are reaching the EU market now. Two products for treatment of joint disease in horses have received positive opinions and will present an innovative option in the veterinary field. This should be acknowledged as a major achievement by the companies behind the products, who made it through the EU authorisation process despite limited published guidance and regulatory experience.

Stem cells are non-terminally differentiated, self[1]renewing cells that harbour the ability to produce mature, differentiated daughter cells. They serve to regulate or participate in normal tissue homeostasis and embryonic and foetal development. They can be harvested from a variety of tissues, e.g. bone marrow, umbilical cord, blood, fat, or embryos, and subsequently produced in a manufacturing facility. Although stem cells share the same principal characteristics of self-renewal potential and differentiation, stem-cell-based medicinal products do not constitute a homogeneous class.

Stem cell therapy is intended for mitigating, treating, or preventing disease in animals or humans. They hold considerable promise for therapeutic applications in various conditions, including metabolic, degenerative and inflammatory diseases, and for the repair and regeneration of damaged or lost tissues. The stem cells now produced as veterinary medicines are allogeneic stem cells, i.e. those in which the cells are collected from a donor animal and used in a recipient animal of the same species. Stem cell treatments may lower symptoms of the disease or condition that is being treated, which may also allow reduce the drug intake.

The first human stem cell product in the EU, Holoclar, was designated as an orphan medicine and recommended for conditional approval in 2014. This allowed the EMA to provide support including free scientific advice during Holoclar’s development. Holoclar is used in the eye to replace damaged cells on the surface (epithelium) of the cornea, the transparent layer in front of the eye covering the iris, to treat moderate to severe limbal stem cell deficiency due to physical or chemical burns to the eye.

In June 2018, the first veterinary stem cell product, Arti[1]Cell Forte, received a positive opinion from the EMA, followed by an EU marketing authorisation in spring 2019. Arti-Cell Forte contains chondrogenic induced equine allogeneic peripheral blood-derived mesenchymal stem cells as the active substance and is given as a single injection into an affected joint for reduction of mild to moderate lameness linked to joint inflammation in horses. The stem cells are treated so that they develop towards cartilage cells, which can assist in repairing damaged cartilage in the joint and aims to activate chondroprotective mechanisms, such as producing extracellular matrix and influencing the inflammatory process in the joint.