EU/3/18/2003 - orphan designation for treatment of Lassa fever

ribavirin

Overview

On 21 March 2018, orphan designation (EU/3/18/2003) was granted by the European Commission to Pharmadev Healthcare Ltd, Ireland, for ribavirin for the treatment of Lassa fever.

The sponsor’s address was updated in April 2021.

What is Lassa fever?

Lassa fever is a viral infection that causes symptoms such as fever, headache, sore throat, muscle, belly and chest pain, nausea, vomiting, diarrhoea, cough, face swelling, low blood pressure and bleeding.

The disease is caused by the Lassa virus and is transmitted to people mostly by contact with urine or blood of infected Mastomys rats.

Lassa fever is debilitating and life-threatening, with 1 to 2% of people who become infected dying from it.

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

At the time of designation, Lassa fever affected less than 0.001 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 50 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).

---

*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 the orphan designation there were no medicines or vaccine authorised for Lassa fever in the EU. Symptoms of the disease were managed with painkillers, transfusion with blood products and fluids.

How is this medicine expected to work?

Ribavirin is an antiviral medicine that is already authorised for treating another viral infection, chronic hepatitis C. The medicine is known as a 'nucleoside analogue' and is thought to work by interfering with the virus genetic material, which viruses need to survive and multiply.

What is the stage of development of this medicine?

The effects of ribavirin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with ribavirin in patients with Lassa fever were ongoing.

At the time of submission, ribavirin was not authorised anywhere in the EU for Lassa fever 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 15 February 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/3/18/2003: Public summary of opinion on orphan designation: Ribavirin for the treatment of Lassa fever

Adopted Reference Number: EMA/132080/2018

Key facts

• Active substance:
  ribavirin
• Intended use:
  Treatment of Lassa fever
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2003
• Date of designation:
  21/02/2018
• Sponsor:

  Pharmadev Healthcare Ltd

Review of designation

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