EU's First Stem Cell Treatment Recommended by EMA
Posted 07 January 2015 | By
A major milestone in stem cell therapy was reached in December 2014 when a European Medicines Agency (EMA) committee recommended the conditional approval of Holoclar, the first stem-cell product to be recommended for approval in the EU.
Holocloar, developed by Chiesi Farmaceutici S.p.A. and Holostem Terapie Avanzate, is an advanced-therapy medicinal product (ATMP) granted orphan designation for limbal stem cell deficiency (LSCD) caused by chemical or physical burns.
LSCD is a rare condition that causes ocular pain, decreased vision, and sometimes blindness. LSCD affects only 3.3 out of 100,000 people in the EU, far below the 5 per 10,000 threshold for orphan product designation set in (EC) No 141/2000. Holoclar was given orphan product designation on 7 November 2008, which allowed Chiesi to be given scientific advice and protocol assistance at no cost during the product’s development.
Recommended for Approval
On 19 December 2014, EMA's Committee for Medicinal Products for Human Use (CHMP), which is charged with recommending whether drug products should be approved in the EU, recommended Holoclar be granted conditional approval. The decision now awaits confirmation by the European Commission.
Holoclar is the first product recommended in the EU for approval to treat LSCD, which both the Committee for Advanced Therapies (CAT) and CHMP considered when recommending Holoclar for conditional approval. The recommendation for conditional approval of Holoclar is based on data from retrospective studies; full approval will require additional study and more comprehensive data.
Stem cells are a type of cell found in the body that are unspecialized and capable of replicating themselves through cell division and can be made to become other types of tissue with specific function. These traits have made stem cells a focus of research into regenerative medicine.
All stem cell medicines in the EU are required to be reviewed by EMA through its centralized procedure, which allows a product to be authorized for use throughout the EU. All applications for marketing authorization for stem cell products are reviewed by both CAT and CHMP.
EMA Recommendation of Holoclar