First stem-cell therapy recommended for approval in EU

New treatment for rare condition caused by burns to the eye

The European Medicines Agency (EMA) has recommended Holoclar, the first advanced therapy medicinal product (ATMP) containing stem cells, for approval in the European Union (EU). Holoclar is a treatment 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 recommendation was made by the Committee for Medicinal Products for Human Use (CHMP) based on a robust assessment carried out by the Committee for Advanced Therapies (CAT), the Agency’s expert committee for ATMPs.

‘This recommendation represents a major step forward in delivering new and innovative medicines to patients,’ says Enrica Alteri, Head of EMA’s Human Medicines Evaluation Division. ‘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. This allowed the Agency to provide support including several rounds of free scientific advice to the applicant during Holoclar’s development

Fonte: EMA Europe