First stem-cell therapy recommended for approval in EU

By William Chin, PhD, Scientific Coordinator, EUCRAF

In the last meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) of the year 2014, Holoclar, the first advanced therapy medicinal product (ATMP) containing stem cells, has been recommended for approval in the EU. Holoclar is a treatment for moderate to severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eye(s) in adults. The recommendation was made based on the assessment by the Committee for Advanced Therapies (CAT). This ATMP has been designated as an orphan medicine. ATMPs consists of innovative medicines that are intended for gene therapy, cell therapy or tissue engineering. Holoclar is a living tissue equivalent intended to be transplanted in the affected eye(s) after removal of the altered corneal epithelium. It is made from a biopsy taken from a small undamaged area of the patient’s cornea and grown in the laboratory using cell culture. The applicant for Holoclar is Chiesi Farmaceutici S.p.A.


1. EMA Press Release, 19 Dec 2014, First stem-cell therapy recommended for approval in EU