First stem cell-based medicinal product approved in Europe

The European Commission has granted a conditional marketing authorization to Chiesi's Holoclar, an advanced therapy based on autologous stem cells capable of restoring the eyesight of patients with severe cornea damage. Holoclar is the first medicinal product based on stem cells to be approved and formally registered in the Western world.

The active substance of Holoclar is ex-vivo expanded autologous human corneal epithelial cells containing stem cells, an ophthalmological product that acts by replacing damaged corneal cells, including limbal stem cells responsible for continuous regeneration and maintenance of the corneal epithelium. By re-establishing a reservoir of stem cells in the eye, Holoclar initiates normal corneal cell growth and maintenance. Holoclar, which looks like a contact lens, is able to repair the damaged ocular surface, improve or resolve symptoms of pain, photophobia and burning, and improve the patient's visual acuity without causing any rejection because it consists of the patient's own cells.

The approved indication is treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularization in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. A minimum of 1-2 mm2 of undamaged limbus is required for biopsy. The therapy received orphan drug designation in 2008 and was recommended for approval in December 2014. Holoclar is manufactured by Holostem Advanced Therapies, a spin-off of the University of Modena and Reggio Emilia at the Centre for Regenerative Medicine "Stefano Ferrari" (CMR) of the same University (Chiesi Farmaceutici News Release; EMA Web site).