First stem-cell therapy recommended for approval in EU by the European Medicines Agency

Written by Elena Conroy

The first advanced therapy containing stem cells for a rare eye condition has received a positive assessment from the European Medicines Agency (EMA)

The EMA recommended Holoclar, developed by the Italian pharmaceutical company Chiesi, which aims to treat limbal stem cell deficiency (LSCD). LSCD is a rare eye condition affecting 3.3 out of 100,000 people in the European Union (EU). and normally occurs after damage from physical or chemical burns to the eye.

Cells are normally stripped off the surface of the eye daily by blinking and contact with air which are subsequently replaced by a population of limbal stem cells located between the cornea and the sclera (white of the eye).However, if these stem cells are damaged the lost cells can no longer be replaced which cause the eye to scar ultimately leading to blindness.

The EMA’s recommendation came from the Committee for Medicinal Products for Human Use and was based on a ‘robust assessment’ of the clinical and research data.

“This is exciting both because there are no other treatments for this sort of damage to the eye and because this is the first example in which a ‘medicine’ based on stem cells has been approved for use in humans,” explained Professor Graham McGeown from Queen’s University Belfast.

Holoclar is made from a biopsy taken from a small undamaged area (minimum of 1-2 mm2) of the patient’s cornea and grown in the laboratory using cell culture. The cultured limbal stem cells are subsequently transplanted in the affected eye(s) after the removal of the damaged corneal epithelium.

“This is a therapy that has been done a lot and is very successful, we’ve treated around 20 people at Moorfields hospital,” stated Prof Chris Mason from University College London.

Holoclar could offer an alternative to transplantation for replacing corneal epithelium and may also be used in combination to increase the chances of a successful corneal transplant. Another important advantage is the reduced risk of rejection since the cells are autologous.

‘I hope that Holoclar is not just an exception and that many more cellular therapy products will see market in the next year’ stated Dusko Ilic from King’s College London.

The recommendation will now be sent to the European Commission, who will decide whether to award an EU-wide marketing authorisation. Once marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State taking into account the potential use of this medicine in the context of the national health system of that country.


EMA press release:

BBC News: