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The first stem-cell medication approved in the EU promotes regeneration and healing to the outer layer of the cornea.
In a landmark step, the European Medicines Agency announced on Dec. 19, 2014 that it recommends approval of Holoclar, a treatment for moderate to severe limbal stem cell deficiency due to injury to the eye. Holoclar is the first product containing stem cells that has received this recommendation.
Limbal stem cells replace cells in the eye that are shed by the act of blinking. After damage to the eye as a result of chemical burns or physical injury, the corneal epithelium is often unable to repair and renew itself. Limbal stem cell deficiency can lead to pain, photobia, and potentially, blindness.
Holoclar is described by EMA as a “living tissue equivalent” to material taken from a donor and its use serves as an alternative to donor transplantation. The advanced therapy medicinal product (ATMP) is made from a biopsy taken from a small, unaffected area of a patient’s corneal epithelium and grown in the lab using cell culture. Use of Holoclar has also been shown to increase the chances of successful corneal implant, as the limbal stem cells in the medicinal product help promote cell regeneration and healing in the eye.
Use of the product reduces the risk of rejection compared with transplanting tissue from a donor and does not require surgery on a patient’s healthy eye, unlike current standard procedures to treat limbal stem cell deficiency of the eye.
"This recommendation represents a major step forward in delivering new and innovative medicines to patients,” said Enrica Alteri, head of EMA’s Human Medicines Evaluation Division, in a press release. “EMA has used all available support tools to facilitate the development and assessment of Holoclar. It is an advanced therapy medicinal product that has been designated as an orphan medicine.”
The EMA recommendation does not guarantee approval of the ATMP by the agency, but is usually a good indicator of an approval. The Committee for Medicinal Products for Human Use and the Committee for Advanced Therapies recommended conditional marketing authorization for the ATMP and said that because the data were based on retrospective studies, an additional study on the use of Holoclar should be conducted.