EU/3/15/1512 - orphan designation for treatment of avian influenza

Anti-H5N1 equine immunoglobulin F(ab')2 fragments

Overview

On 28 July 2015, orphan designation (EU/3/15/1512) was granted by the European Commission to Fab'entech, France, for anti-H5N1 equine immunoglobulin F(ab')₂ fragments for the treatment of avian influenza.

What is avian influenza?

Avian influenza (or bird flu) is an infectious disease of birds caused by certain subtypes of the influenza A virus. Avian influenza has rarely spread to humans through contact with infected poultry. To date, the subtype known as H5N1 has caused serious human infections; a very few cases in people have also been reported with another subtype, H7N9.

Avian influenza can be life threatening as it can lead to severe pneumonia (infection of the lungs), problems with breathing and multi-organ failure.

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

At the time of designation, avian influenza affected less than 0.01 in 10,000 people in the European Union (EU)*. This is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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, antiviral medicines such as oseltamivir were authorised in the EU to treat influenza infections including avian influenza.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with avian influenza because early studies in experimental models indicate that it might improve survival. 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?

This medicine is made of antibody fragments. Antibodies are proteins that attach to a specific target in the body and can help to fight infections and other diseases. The antibody fragments in the medicine are expected to attach to the H5N1 virus and neutralise its ability to infect body cells.

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 avian influenza were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for avian influenza. Orphan designation of the medicine had been granted in Australia for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 June 2015 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/3/15/1512: Public summary of opinion on orphan designation: Anti-H5N1 equine immunoglobulin F(ab')2 fragments for the treatment of avian influenza

AdoptedReference Number: EMA/COMP/433479/2015

Key facts

• Active substance:
  Anti-H5N1 equine immunoglobulin F(ab')2 fragments
• Intended use:
  Treatment of avian influenza
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/15/1512
• Date of designation:
  28/07/2015
• Sponsor:

  Fab'entech; 24 rue Jean Baldassini; 69007 Lyon; France; Tel. [phone]; Fax [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.