EU/3/14/1340 - orphan designation for treatment of limbal stem cell deficiency

Cultured allogeneic corneal limbal stem cells

Overview

On 15 October 2014, orphan designation (EU/3/14/1340) was granted by the European Commission to NHS National Services Scotland, trading as Scottish National Blood Transfusion Service, United Kingdom, for cultured allogeneic corneal limbal stem cells for the treatment of limbal stem cell deficiency.

What is limbal stem cell deficiency?

Limbal stem cell deficiency is an eye condition in which the patient lacks cells called limbal stem cells, which are found at the edge of the cornea (the transparent layer in front of the eye) and which continuously renew and repair the cornea. The deficiency of limbal stem cells leads to clouding of the cornea and may result in impaired vision or blindness.

Limbal stem cell deficiency is a long-term disease that is debilitating to patients because of the impaired vision and discomfort it causes.

What is the estimated number of patients affected by the condition?

At the time of designation, limbal stem cell deficiency affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, there were no satisfactory treatments authorised for treating the condition in the EU. Treatment being used included transplantation of limbal stem cells, which were obtained either from a donor or where possible from the patient's other eye.

How is this medicine expected to work?

This product is made of limbal stem cells that are taken from a donor's eye and grown on a membrane in the laboratory. The membrane is then implanted into the patient's damaged eye or eyes. The donor stem cells grown on the membrane are then expected to help the cornea to regenerate, restoring the patient's vision.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with limbal stem cell deficiency were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for limbal stem cell deficiency or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 September 2014 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/14/1340: Public summary of opinion on orphan designation: Cultured allogeneic corneal limbal stem cells for the treatment of limbal stem cell deficiency

AdoptedReference Number: EMA/COMP/557523/2014

Key facts

• Active substance:
  Cultured allogeneic corneal limbal stem cells
• Intended use:
  Treatment of limbal stem cell deficiency
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1340
• Date of designation:
  15/10/2014
• Sponsor:

  NHS National Services Scotland Trading as Scottish National Blood Transfusion Service; 21 Ellen's Glen Road; Edinburgh EH17 7QT; United Kingdom; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.