EU/3/08/606 - orphan designation for treatment of Behçet's Disease

recombinant human residue 41 glutamic acid to glutamine variant of interferon alfa-2b

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in March 2011 on request of the sponsor.

On 19 January 2009, orphan designation (EU/3/08/606) was granted by the European Commission to Creabilis Therapeutics S.p.A., Italy, for recombinant human residue 41 glutamic acid to glutamine variant of interferon-alfa-2b for the treatment of Behçet's disease.

What is Behçet's disease?

Behçet's disease is an autoimmune disease (a disease in which the immune system, the body's natural defence, attacks parts of the body). In Behçet's disease, the immune system attacks blood vessels throughout the body. The exact cause of the disease is unknown. As a result of the damage to the blood vessels, almost all patients develop signs such as painful sores in the mouth called aphthous ulcers. They can also develop sores on the genitals, inflammation inside the eye and skin problems. The inflammation inside the eye can affect the uvea (the middle part of the eye), the iris or the retina (the light-sensitive surface at the back of the eye) and can lead to blurred vision, pain, and redness. Behçet's disease is a debilitating and long-lasting disease because of the sores in the mouth and on the genitals and inflammation of the blood vessels throughout the body.

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

At the time of designation, Behçet's disease affected less than 1 in 10,000 people in the European Union (EU)*. This is below the threshold for orphan designation which is 5 in 10,000 people, and is equivalent to a total of around 50,000 people. 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 based on data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are available?

At the time of designation, there were no treatments that could cure Behçet's disease. Treatment focussed on relieving the symptoms of the disease, reducing discomfort and preventing serious complications. Commonly used treatments included medicines that reduce inflammation, such as steroids. Colchicine was approved for Behçet's disease in one Member State.

The sponsor has provided sufficient information to show that recombinant human residue 41 glutamic acid to glutamine variant of interferon-alfa-2b might be of significant benefit for the patients because it may improve the treatment of ocular symptoms. 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?

Recombinant human residue 41 glutamic acid to glutamine variant of interferon-alfa-2b is a modified type of a type of interferon. Interferons are natural substances produced by the body to help it fight against attacks such as infections caused by viruses. Several types of interferon are used as medicines to treat diseases such as cancer and hepatitis C. Interferons are also used in some diseases to reduce the activity of the immune system, including Behçet's disease although they are not authorised for use in this disease.

This medicine is a modified form of interferon-alfa-2b, in which one of the amino acids that make up the protein has been exchanged for another. This modification is expected to make the medicine more resistant to breakdown in the body, so that lower doses can be given to the patient.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, one clinical trial had been performed in healthy volunteers.

At the time of submission, recombinant human residue 41 glutamic acid to glutamine variant of interferon-alfa-2b was not authorised anywhere in the world for Behçet's disease 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 5 November 2008 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• he seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• and either the rarity of the condition (affecting not more than 5 in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/08/606: Public summary of positive opinion for orphan designation of recombinant human residue 41 glutamic acid to glutamine variant of interferon-alfa-2b for the treatment of Behà§et’s disease

Adopted Reference Number: EMEA/COMP/612595/2008

Key facts

• Active substance:
  recombinant human residue 41 glutamic acid to glutamine variant of interferon alfa-2b
• Intended use:
  Treatment of Behçet's Disease
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/08/606
• Date of designation:
  19/01/2009
• Sponsor:

  Creabilis Therapeutics S.p.A.; Bioindustry Park del Canavese; Via Ribes 5; 10010 Colleretto Giacosa (TO); Italy; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]