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Date: 2014-12-19

Type of information: Positive opinion for the granting of a Market Authorisation in the EU

Product name: Holoclar®

Compound: ex-vivo expanded autologous human corneal epithelial cells containing stem cells

Therapeutic area: Ophtalmological diseases - Rare diseases

Action mechanism:

Limbal stem cells are located in the eye at the border between the cornea (clear front part of the eye) and the sclera (white of the eye). These cells are important for regenerating and healing damage to the outer layer of the cornea (corneal epithelium). Physical or chemical burns can cause loss of these stem cells, resulting in LSCD, a condition that is estimated to affect about 3.3 out of 100,000 people in the European Union. Symptoms include pain, photophobia (painful sensitivity to light), inflammation, corneal neovascularisation (excessive ingrowth of blood vessels into the cornea), loss of corneal transparency, and eventually blindness. 

Holoclar® is a living tissue equivalent intended to be transplanted in the affected eye(s) after removal of the altered corneal epithelium. It is made from a biopsy taken from a small undamaged area (minimum of 1-2 mm2) of the patient’s cornea and grown in the laboratory using cell culture. Holoclar® can offer an alternative to transplantation for replacing altered corneal epithelium in some cases, and it has been shown to increase the chances of a successful corneal transplant where the injury has caused extensive eye damage. It reduces the risk of rejection compared with transplanting tissue from a donor and does not require surgery on the patient’s other eye as only a small biopsy is performed to collect the cells, thus reducing the risk of damage to the healthy eye. Therefore, Holoclar® may also be suitable where both eyes are affected by moderate to severe LSCD.

Company: Chiesi Farmaceutici (Italy)

Disease:

 moderate to severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eyes in adults

Latest news:

* On December 19, 2014, the CHMP has recommended granting a conditional marketing authorisation for the orphan medicine Holoclar® for the treatment of moderate to severe limbal stem cell deficiency due to physical or chemical burns to the eyes in adults. Holoclar® is the first stem-cell therapy recommended for approval in EU. 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. The CAT and the CHMP considered that Holoclar® provided a first treatment option for this rare eye condition and recommended a conditional marketing authorisation because, although the data supplied by the applicant show that the medicine\'s benefits outweigh its risks, the data are based on retrospective studies and are not yet comprehensive. Therefore, an additional study on the use of Holoclar® should be conducted.
Holoclar® was designated as an orphan medicine and an advanced therapy medicinal product (ATMP) and EMA provided protocol assistance to the applicant during the development of the medicine on multiple occasions. Both orphan and ATMP designations and the associated incentives, such as free scientific advice or protocol assistance, are among the Agency’s most important instruments to encourage the development of medicines for patients suffering from rare diseases, and the development of innovative medicinal products.

Patents:

Submission of marketing authorization application USA :

Submission of marketing authorization application UE:

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization:

UE authorization:

Favourable opinion UE: 2014-12-19

Favourable opinion USA:

Orphan status USA:

Orphan status UE: 2008-11-07

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes