EU/3/14/1294

On 29 July 2014, orphan designation (EU/3/14/1294) was granted by the European Commission to Dr Ulrich Granzer, Germany, for sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH for the treatment of necrotising soft tissue infections.

The sponsorship was transferred to FGK Representative Service GmbH, Germany, in March 2015.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What are necrotising soft tissue infections?

Necrotising soft tissue infections are severe and rapidly progressing bacterial infections that destroy skin, muscle, and other soft tissues. A necrotising infection causes areas of tissue to die and often leads to failure of several organs, which eventually may lead to death. The most dangerous type of these infections is commonly known as ‘flesh-eating disease’.

Necrotising soft tissue infections are life threatening and long-term debilitating because of the extent of damage to soft tissues and high mortality rate.

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

At the time of designation, necrotising soft tissue infections affected less than 1 people in 10,000 in the European Union (EU). This was equivalent to fewer than 51,000 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).

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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. At the time of designation, this represented a population of 512,900,000 (Eurostat 2014).

 

What treatments are available?

At the time of designation, antibiotics were authorised in the EU to treat necrotising soft tissue infections. Surgery (often repeated) and admission to critical care units were also used to try to control the infections.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with necrotising soft tissue infections because it works in a different way to existing treatments and early studies show that it might improve the outcome of patients with the condition. 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?

Necrotising soft tissue infections can produce rapid organ failure due to a massive inflammatory response affecting the whole body. This inflammatory response is thought to be caused by an excess production of cytokines (substances produced by the body as a defence mechanism, for example against infections) and of toxins from bacteria.

This medicine is made of a small peptide that mimics part of a receptor called CD28, which is found on the surface of some white blood cells and is involved in their activation. Although its mechanism of action is not fully understood, the medicine is expected to block the excessive production of cytokines by white blood cells, thus improving the outcome of patients with necrotising soft tissue infections.

What is the stage of development of this medicine?

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 the medicine in patients with necrotising soft tissue infections had been completed and further studies were planned.

At the time of submission, the medicine was not authorised anywhere in the EU for necrotising soft tissue infections. Orphan designation of the medicine had been granted in the United States for necrotising soft tissue infections.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 June 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.

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