EU/3/15/1568

On 11 November 2015, orphan designation (EU/3/15/1568) was granted by the European Commission to NV Hemispherx BioPharma Europe, Belgium, for interferon alfa-n3 for the treatment of Middle East respiratory syndrome.

What is Middle East respiratory syndrome?

Middle East respiratory syndrome is an infection caused by a virus called Middle East respiratory syndrome coronavirus. It usually affects the lungs and airways with fever, cough and shortness of breath. Patients may also get diarrhoea, vomiting and muscle pain.

Middle East respiratory syndrome is a severe and life-threatening illness because it can cause respiratory failure that requires mechanical ventilation and support in an intensive-care unit. Some patients may develop organ failure, especially of the kidneys, or septic shock (life-threatening drop in blood pressure).

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

At the time of designation, Middle East respiratory 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).

---

*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?

No satisfactory methods for treating Middle East respiratory syndrome were authorised in the EU at the time of designation. Management involved treatment of individual symptoms, with measures to support the working of vital organs in severe disease.

How is this medicine expected to work?

This medicine contains a type of interferon called interferon alfa-n3. Interferons are natural substances produced by the body that have a number of actions; one of these is to help fight off viruses. The Middle East respiratory syndrome virus overcomes the body’s natural defences by stopping the body’s own interferons from working properly. It is expected that giving additional interferon in the form of interferon alfa-n3 will help to restore the body’s defence against the virus.

What is the stage of development of this medicine?

The effects of interferon alfa-n3 have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with interferon alfa-n3 in patients with Middle East respiratory syndrome had been started.

At the time of submission, interferon alfa-n3 was not authorised anywhere in the EU for Middle East respiratory 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 8 October 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.

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