EU/3/19/2173 - orphan designation for treatment of acute radiation syndrome

Imidazolyl ethanamide pentandioic acid

Overview

On 28 June 2019, orphan designation EU/3/19/2173 was granted by the European Commission to Myelo Therapeutics GmbH, Germany, for imidazolyl ethanamide pentandioic acid (also known as Myelo001) for the treatment of acute radiation syndrome.

The sponsor’s address was updated in April 2020.

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 radiation in a very short period of time. 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. 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 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 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 518,400,000 (Eurostat 2019).

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 exact way the medicine works is not clear but it is thought to affect the activity of a protein in the body called NFkB. When the body is exposed to radiation, NFkB causes release of substances that affect the production of blood cells. This medicine is expected to block the activity of NFkB and as a result slow down the destruction of blood cells and reduce the time it takes for the production of blood cells to recover. 

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

For ethical reasons, clinical trials with the medicine in patients with acute radiation syndrome were not planned, but clinical trials were conducted in patients with low white blood cell count who had been treated with cancer medicine or had received radiotherapy.
At the time of submission, imidazolyl ethanamide pentandioic acid was not authorised anywhere in the EU for the treatment of acute radiation syndrome. Orphan designation of the medicine had been granted in the United States for the treatment of acute radiation syndrome.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 23 May 2019, 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/3/19/2173: Public summary of opinion on orphan designation: Imidazolyl ethanamide pentandioic acid for the treatment of acute radiation syndrome

Adopted Reference Number: EMADOC-628903358-1036

Key facts

• Active substance:
  Imidazolyl ethanamide pentandioic acid
• Intended use:
  Treatment of acute radiation syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/19/2173
• Date of designation:
  28/06/2019
• Sponsor:

  Myelo Therapeutics GmbH; Rheinsberger Strasse 7; 10115 Berlin; Deutschland; Tel. [phone]; E-mail: [e-mail]