EU/3/18/1985 - orphan designation for treatment of acute radiation syndrome

rusalatide acetate

Overview

On 22 February 2018, orphan designation (EU/3/18/1985) was granted by the European Commission to Raremoon Consulting Ltd, United Kingdom, for rusalatide acetate (also known as TP508) for the treatment of acute radiation syndrome.

The sponsorship was transferred to Raremoon Consulting Esp S.L., Spain, in May 2020.

What is acute radiation syndrome?

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

Symptoms can begin within a few hours of 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 of radiation can result in effects on the central nervous system (brain and spinal cord) and rapidly lead to death.

Acute radiation syndrome is a life-threatening condition because it can lead to damage 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 less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer 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 517,400,000 (Eurostat 2018).

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?

The medicine mimics the action of thrombin, one of the substances involved in stimulating tissue repair after injury. In laboratory studies the medicine has been shown to help cells repair breaks in their DNA caused by radiation, as well as stimulating the growth of blood vessels, reducing inflammation and stimulating stem cells for tissue repair. These actions are expected to help reduce the damage caused by radiation.

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. Orphan designation of the medicine had been granted in the United States for acute radiation syndrome.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 January 2018 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/3/18/1985: Public summary of opinion on orphan designation: Rusalatide acetate for the treatment of acute radiation syndrome

Adopted Reference Number: EMA/52859/2018

Key facts

• Active substance:
  rusalatide acetate
• Intended use:
  Treatment of acute radiation syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/1985
• Date of designation:
  22/02/2018
• Sponsor:

  Raremoon Consulting Esp S.L.;