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Europe gains access to first stem cell drug

Chiesi's Holoclar has been given the green light for a rare eye condition

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The Western world's first stem cell derived drug has been approved by the European Commission. 

Chiesi's Holoclar (an ex-vivo expanded autologous human corneal epithelial cells containing stem cells), has been approved for moderate to severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eye(s) in adults.

It is the first medicine recommended for LSCD, a rare eye condition that can result in blindness. 

The EMA has recently given the medicine orphan drug status, extending its patent life in Europe (although it may find itself immune to attempts to copy it), with its safety and efficacy arm the CHMP advising its approval in December last year. 

The recommendation by the EMA was based on an assessment carried out by the Committee for Advanced Therapies (CAT), the EMA's expert committee for advanced therapy medicinal product (ATMPs).

But even though the CAT and the CHMP both believe this drug to be safe and effective, they both suggested that it be approved under a conditional marketing authorisation.

This is because Chiesi's submission data was based on retrospective studies, and is not yet comprehensive - although it did still show that the medicine's benefits outweigh its risks.

However, the EMA said an additional study on the use of Holoclar should be conducted before a full licence can be issued. 

The Commission has taken these issues into consideration, and has said its approval is conditional on the Italian pharma firm conducting these additional studies, but will allow it to be marketed in the EU while the studies are ongoing.  

Professor Michele De Luca, scientific director and co-founder of Holostem, as well as Director of the CMR of the University of Modena, both of which helped the firm develop this drug, said: “The authorisation process has been long and complex, but the result achieved shows that cells can be cultured according to pharmaceutical standards appropriate to guarantee safety and efficacy.

“In addition, in a period of great confusion about the real therapeutic possibilities of stem cells, such as the one we are living in, being able to demonstrate that stem cells can be definitely safe and successful in a controlled clinical setting is more important than ever.”

Living tissue

Stem cells can act as a repair system for the body and limbal stem cells are located in the eye at the border between the cornea (clear front part of the eye) and the sclera (white of the eye).

These cells are important for regenerating and healing damage to the outer layer of the cornea (corneal epithelium). Physical or chemical burns can cause loss of these stem cells, resulting in LSCD, a condition that is estimated to affect just 3.3 out of 100,000 people in the European Union.

Article by
Ben Adams

23rd February 2015

From: Sales, Regulatory



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