EU/3/15/1596: Orphan designation for the treatment of partial deep dermal and full-thickness burns

Partial deep dermal and full thickness burns are deep burns to the skin. Full thickness burns can also involve the layers of fat beneath the skin.

These types of burns are debilitating in the long term because of the scarring, disfigurement and disability that can follow. They are also life threatening because they can lead to serious infection.

At the time of designation, partial deep dermal and full thickness burns affected less than 2.9 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 149,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

* 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 512,900,000 (Eurostat 2015).

At the time of the orphan designation, some products were authorised in the EU for wound care in patients with deep burns (e.g. povidone-iodine) and for removing dead tissue (e.g. NexoBrid).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with partial deep dermal and full thickness burns. The product works in a different way to currently authorised treatments and early studies show that the medicine could be effective as a skin graft. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is a graft containing cells known as fibroblasts and keratinocytes obtained from the patient's skin. Fibroblasts are normally present in the dermis, the lower layer of the skin, while keratinocytes are abundant in the epidermis, the upper layer. Both types of cells are arranged within a protein gel, with the fibroblasts within and the keratinocytes on top to form a product that mimics the skin and can be surgically implanted over the burn wound.

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

At the time of submission of the application for orphan designation, a clinical trial with this medicine including patients with partial deep dermal and full thickness burns was ongoing.

At the time of submission, the product was not authorised anywhere in the EU for partial deep dermal and full thickness burns 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 12 November 2015 recommending the granting of this designation.

• 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.