EU/3/06/380 - orphan designation for treatment of alopecia universalis

Diphenylcyclopropenone

Overview

Please note that this product was withdrawn from the Community register of designated orphan medicinal products in July 2013 on request of the sponsor.

On 29 June 2006, orphan designation (EU/3/06/380) was granted by the European Commission to Orfagen, France, for diphenylcyclopropenone for the treatment of alopecia universalis.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is alopecia universalis?

Alopecia universalis is a disease characterised by complete hair loss of the whole body. It affects patients in an acute form (loss of hair within weeks) or in a slower form (loss of hair can progress for up to two years). Usually patients do not recover from the hair loss and the condition becomes chronic. Alopecia universalis can affect patients of both sexes and of any age. Patients suffering alopecia universalis 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 universalis 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 117,000 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).

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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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

What treatments are available?

No satisfactory methods were authorised at the time of application.

How is this medicine expected to work?

Hair loss seen in alopecia universalis 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 sensitisation, which is an allergic reaction to oneself. 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 for hair growth locally.

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 universalis had been completed. The sponsor of the application planned to conduct further clinical studies.

Diphenylcyclopropenone was not authorised anywhere worldwide for alopecia universalis or designated as an 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.

EU/3/06/380: Public summary of positive opinion for orphan designation of diphenylcyclopropenone for the treatment of alopecia universalis

Adopted Reference Number: EMEA/COMP/428296/2006 Rev.2

Key facts

• Active substance:
  Diphenylcyclopropenone
• Intended use:
  Treatment of alopecia universalis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/06/380
• Date of designation:
  29/06/2006
• Sponsor:

  Orfagen; CRDPF Langlade; 3 Avenue Hubert Curien - BP 13562; 31035 Toulouse Cedex 1; France; Tel. [phone]; Fax [phone]; E-mail [e-mail]