EU/3/06/379

On 29 June 2006, orphan designation (EU/3/06/379) was granted by the European Commission to Orfagen, France, for diphenylcyclopropenone for the treatment of alopecia totalis.

What is alopecia totalis?

Alopecia totalis is a disease characterised by massive hair loss (more than 90%) of the scalp. It affects patients in an acute form (loss of hair within weeks) or in a slower form (hair loss that can progress for up to two years). Usually patients do not recover form the hair loss and the condition becomes chronic. Alopecia totalis can affect patients of both sexes and of any age. Patients suffering alopecia totalis report psychological consequences (distress, sadness) that are particularly severe in children and women.

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

At the time of designation, alopecia totalis affected not more than 2.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 115,000 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 based on data from the European Union (EU 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

No satisfactory methods exist that were authorised at the time of application.

How is this medicine expected to work?

Hair loss seen in alopecia totalis is thought to be caused by cells of the immune system (body’s own defence mechanism against infection and disease) attacking the hair follicles. Diphenylcyclopropenone can act as local irritant and trigger local sensitization, which is an allergic reaction to itself. By doing this, it is thought to mount an immune response and produce populations of immune cells that oppose the action of the autoreactive cells that destroy hair loss. This way it is thought to allow hair growth locally. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with alopecia totalis were completed. The sponsor of the application plans to conduct further clinical studies.

Diphenylcyclopropenone was not authorised anywhere worldwide for alopecia totalis or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 May 2006 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 Community) 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.