EU/3/16/1709

On 14 July 2016, orphan designation (EU/3/16/1709) was granted by the European Commission to Hypo-Stream Ltd, United Kingdom, for sodium hypochlorite for the treatment of partial deep dermal and full thickness burns.

What are partial deep dermal and full thickness burns?

Partial deep dermal and full thickness burns are deep burns to the skin. In the case of full thickness burns, injury can also extend to 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.

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

At the time of designation, partial deep dermal and full thickness burns affected approximately 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 77,000 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).

---

* 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 513,700,000 (Eurostat 2016).

What treatments are available?

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, with early studies showing improved survival in patients with widespread burns. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

When applied to the burn wound, sodium hypochlorite causes a reduction in the amount of proteins known as cytokines, which are involved in inflammation. The lower levels of cytokines and the subsequent reduction in inflammation are then expected to help in wound healing.

What is the stage of development of this medicine?

The effects of sodium hypochlorite have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with sodium hypochlorite in patients with partial deep dermal and full thickness burns were ongoing.

At the time of submission, a sodium hypochlorite solution was authorised in Italy for disinfection and cleaning of damaged skin (wounds, sores, burns, etc.) and the disinfecting of external genitalia.

At the time of submission, sodium hypochlorite 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 16 June 2016 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.

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