EU/3/10/771 - orphan designation for treatment of low-flow priapism

abarelix

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in October 2012 on request of the sponsor.

On 20 September 2010, orphan designation (EU/3/10/771) was granted by the European Commission to Speciality European Pharma Ltd, United Kingdom, for abarelix for the treatment of low-flow priapism.

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

What is low-flow priapism?

Priapism is the prolonged and often painful erection of the penis occurring without sexual stimulation. In low-flow priapism, the prolonged erections are caused by a blockage of the blood flow out of the penis. This leads to the build-up of blood that is low in oxygen and, as a consequence, the tissues in the penis may not receive enough oxygen. The erections can last for several hours and are usually considered a medical emergency. Low-flow priapism may be caused by a medicine the patient may be taking or a disease of the blood such as sickle-cell disease.

Low-flow priapism is a debilitating disease because it can lead to erectile dysfunction (inability to get or keep an erection) and permanent damage to the penis.

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

At the time of designation, low-flow priapism affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 25,000 people*, and is below the threshold 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

At the time of submission of the application for orphan drug designation, no satisfactory methods were authorised for low-flow priapism in the EU. Treatments used included surgery, medicines known as 'alpha-agonists' that help to widen the veins and treatments for the underlying cause of the priapism if it is known.

How is this medicine expected to work?

The hormone testosterone is involved in causing erections. Abarelix is a gonadotrophin-releasing hormone antagonist, which means that it blocks the effects of a natural hormone called gonadotrophin-releasing hormone (GnRH). GnRH stimulates the testes to produce testosterone. By blocking the actions of GnRH, abarelix is expected to reduce the amount of testosterone in the body, helping to treat episodes of priapism or to prevent them from happening again.

What is the stage of development of this medicine?

The effects of abarelix have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with abarelix in patients with priapism had been started.

At the time of submission, the medicine was authorised in Germany for suppressing testosterone levels in patients with advanced or metastatic hormone-dependent prostate cancer.

At the time of submission, abarelix was not authorised anywhere in the EU for low-flow priapism 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 2 June 2010 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/3/10/771: Public summary of opinion on orphan designation: Abarelix for the treatment of low-flow priapism

AdoptedReference Number: EMA/COMP/411127/2010 Rev. 2

Key facts

• Active substance:
  abarelix
• Intended use:
  Treatment of low-flow priapism
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/10/771
• Date of designation:
  20/09/2010
• Sponsor:

  Speciality European Pharma Ltd; 16 John Street; London; WC1N 2DL; United Kingdom; Tel. + 44 (0)20 7421 7400; Fax + 44 (0)20 7421 7401; 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.