EU/3/15/1535 - orphan designation for treatment of acute radiation syndrome

fibrinogen-coated albumin spheres

Overview

On 10 August 2015, orphan designation (EU/3/15/1535) was granted by the European Commission to Fibreu Limited, United kingdom, for fibrinogen-coated albumin spheres for the treatment of acute radiation syndrome.

The sponsorship was transferred to Fibreu Limitless Research S.L., Spain, in May 2019.

What is acute radiation syndrome?

Acute radiation syndrome (also known as radiation sickness) is a severe illness caused by exposure of the body to a high dose of penetrating radiation in a very short period of time. This can occur, for example, following an accident at a nuclear plant or the use of radioactive material for medical purposes.

Symptoms can begin within a few hours after exposure and their severity depends on the amount of radiation absorbed by the body. Relatively small amounts result in gastrointestinal effects such as nausea (feeling sick), vomiting and diarrhoea, reduction in blood cell count, and tendency to infection and bleeding. Relatively large amounts can result in effects on the central nervous system (brain and spinal cord) and rapid death.

Acute radiation syndrome is a life-threatening disease because it can lead to failure of multiple organs and death.

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

At the time of designation, acute radiation syndrome affected not more than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 500 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. This represents a population of 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of acute radiation syndrome.

How is this medicine expected to work?

Acute radiation syndrome can cause the destruction of the bone marrow, the spongy tissue inside the large bones where blood cells are produced. This causes a reduction in the number of red and white blood cells, as well in the number of platelets, the blood components that help the blood to clot.

This medicine consists of spherical particles coated with fibrinogen, a protein naturally found in the blood that is involved in helping the blood to clot. When the particles reach a bleeding site, the extra fibrinogen provided by the medicine is converted into a fibrous protein known as fibrin, which helps plug the bleeding site, in the same way as a natural blood clot. This additional clotting effect is expected to help counteract any clotting problems and bleeding caused by lack of blood platelets and so reduce the bleeding associated with radiation exposure.

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, no clinical trials with the medicine in patients with acute radiation syndrome had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for acute radiation syndrome 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 July 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/3/15/1535: Public summary of opinion on orphan designation: Fibrinogen-coated albumin spheres for the treatment of acute radiation syndrome

Adopted Reference Number: EMA/COMP/508238/2015

Key facts

• Active substance:
  fibrinogen-coated albumin spheres
• Intended use:
  Treatment of acute radiation syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/15/1535
• Date of designation:
  10/08/2015
• Sponsor:

  Fibreu Limitless Research S.L.; Calle Lopez De Neira Num. 3; Planta 3 Puerta 301; Vigo; Pontevedra 36202; Spain; Tel. [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.