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Rare Diseases: European Approval Of Stem Cell Therapy

March 24, 2015

In December 2014, the European Medicines Agency’s (EMA) Committee for Medicinal Products For Human Use (CHMP) adopts a positive opinion recommending a conditional marketing authorization for the medicinal product, Holoclar, a stem cell therapy for a rare disease. A conditional marketing authorization is given to a product when it fulfils an unmet medical need, when the benefit to public health of immediate availability outweighs the risk – additional clinical data is still required. In February 2015, the European Commission grants a marketing authorization valid throughout the European Union for Holoclar.

Holoclar is for the treatment of the rare eye condition (moderate to severe), Limbal Stem Cell Deficiency (LSCD), in adults due to ocular burns to the eye(s). It is the first medicine recommended for LSCD. Per the EMA website :

“Limbal stem cell deficiency is an eye condition in which the patient lacks cells called limbal stem cells, which are found at the edge of the cornea (the transparent layer in front of the eye) and which continuously renew and repair the cornea. The deficiency of limbal stem cells leads to clouding of the cornea and may result in impaired vision or blindness. Limbal stem cell deficiency is a long-term disease that is debilitating to patients because of the impaired vision and discomfort it causes.”

Holoclar, from Chiesi Farmaceutici S.p.A., is designated an orphan product by the EMA in November 2008. It is a living tissue equivalent, made from a patient’s undamaged area of the cornea and grown in the laboratory using cell culture. It is then transplanted into the affected eye(s) replacing the corneal cells, and results in the start of normal corneal cell growth and maintenance.

An interview, published this month in Nature, with Italian biologist Graziella Pellegrini, describes the 25-year struggle to develop Holoclar.

Please Note: Climbing to Kalindi Mountains of Himalayas, Uttarkashi, Uttarakhand, India” by Sharada Prasad CS from Berkeley, India (Almost There!) [CC BY 2.0] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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