First-Ever Stem Cell Therapy Recommended in EU

Miriam E. Tucker

Disclosures

December 19, 2014

For the first time, a medicinal product containing stem cells has been recommended for approval in the European Union (EU).

On December 19, the European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) recommended the stem cell product Holoclar (Chiesi Farmaceutici S.p.A.) as a first-ever medicinal treatment for severe limbal stem cell deficiency, a condition caused by physical or chemical burns to the eye or eyes in adults, which can result in blindness.

The EMA designated Holoclar as both an orphan medicine and an advanced therapy medicinal product, which enabled the manufacturer to receive free scientific advice and protocol assistance for drug development. The CHMP recommendation was based on an assessment by the expert Committee for Advanced Therapies. Such steps are taken to promote the development of medicines for rare diseases and to encourage innovative medicinal products, according to an EMA statement.

Holoclar replaces damaged limbal stem cells, normally located in the eye between the cornea and the sclera. Limbal stem cell deficiency is estimated to affect about 3.3 per 100,000 people in the EU, causing pain, photophobia, inflammation, corneal neovascularization, loss of corneal transparency, and eventually blindness.

The product is made from tissue taken from an undamaged area of the patient's cornea, grown in cell culture, and transplanted in the affected eye or eyes after removal of the damaged area. In some cases, the treatment offers an alternative to corneal transplantation and thereby reduces the risk for rejection of donor tissue. It may also be suitable in patients with moderate to severe limbal stem cell deficiency in both eyes, according to the EMA statement.

The Committee for Advanced Therapies and the CHMP panels determined that although the product's benefits outweigh its risks, the marketing authorization should be conditional because the data thus far are retrospective and not yet comprehensive. Therefore, the EMA says, "an additional study on the use of Holoclar should be conducted."

The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorization. After that, decisions about price and reimbursement will take place at the level of each member state.

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