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First stem-cell therapy recommended for approval by EU regulator

12 January 2015
Appeared in BioNews 785

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

Holoclar, developed by the Italian pharmaceutical company Chiesi, is aimed at treating limbal stem cell deficiency (LSCD). LSCD is a rare eye condition affecting around three in 100,000 adults in the European Union (EU) and normally occurs after damage from physical or chemical burns to the eye.

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.

Dr Enrica Alteri, head of the human medicines evaluation division at the EMA, said the recommendation was 'a major step forward in delivering new and innovative medicines to patients'.

Expert commentators have also voiced their support for the decision. Professor Graham McGeown from the Centre for Experimental Medicine at Queen's University Belfast, said: '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.'

Cells are normally stripped off the surface of the eye daily by blinking and contact with air. In a healthy eye, these cells are replaced by a population of limbal stem cells located between the cornea (the clear frontal layer of the eye) and the sclera (the white of the eye). However, if these stem cells are damaged, then the lost cells can no longer be replaced. Over time, the eye will begin to scar, leading ultimately to blindness.

Holocar uses a sample from a patient's healthy cornea to extract and grow a sufficient number of limbal stem cells to repair the eye. BBC News says the technique 'works in around 80 percent of cases'. As such it offers an alternative to transplantation for replacing corneal epithelium and may also be used in combination with a corneal transplant to improve the outcome. Additionally, since the cells come from the patient, the risk of rejection is low.

Dr Dusko Ilic, reader in stem cell science, King's College London, commented: 'I hope that Holoclar is not just an exception, and that many more cellular therapy products will see market in the next year.'

The recommendation will now be sent to the European Commission, who will decide whether to award an EU-wide marketing authorisation. Then health authorities from each EU member state will decide if or how they will offer Holoclar as a treatment in their own national health systems.

First stem-cell therapy among seven drugs up for EU nod
PharmaTimes |  19 December 2014
First stem-cell therapy approved for medical use in Europe
The Telegraph |  19 December 2014
First stem-cell therapy recommended for approval in EU
European Medicines Agency (press release) |  19 December 2014
Stem cells: First therapy approved by EU
BBC News |  19 December 2014
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