EU/3/02/122 - orphan designation for treatment of low-flow priapism
Etilefrine
Orphan
Human
Overview
On 13 November 2002, orphan designation (EU/3/02/122) was granted by the European Commission to Laboratoires SERB, France, for etilefrine 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.
Low-flow priapism is a medical disorder in which there is persistent erection of the penis in the absence of sexual interest. Patients with low-flow priapism usually present with a history of several hours of painful erection. It is believed that this condition is caused by obstruction to the normal drainage of blood from the erectile tissues. This obstruction causes build-up of poorly oxygenated blood. If the process continues for several days, abnormal thickening and scarring of the erectile tissue may develop, causing impotence. Low-flow priapism is a serious and debilitating disease if it is not treated adequately and promptly.
At the time of designation, low flow priapism affected approximately 0.11 in 10,000 people in the European Union (EU). This was equivalent to a total of around 4,200 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. At the time of designation, this represented a population of 380,600,000 (Eurostat 2002).
There are no medicinal products that have been authorised for the treatment of priapism in the Community. Treatments which are currently used include medical treatment, for instance with medications consisting of certain adrenaline-like substances (alpha adrenergic agonists) or, if necessary, surgical procedures.
Etilefrine is a substance that stimulates the sympathetic nervous system. This system is involved in regulating several processes such as pulse, blood pressure, and the tone of smooth muscle. The erection of the penis depends on the relaxation of the smooth muscle of the two columns of erectile tissue at either side of the penis (corpora cavernosa). The relaxation causes widening of the blood vessels and the tissue is filled with blood causing the erection. The injection of etilefrine inside the corpora cavernosa leads to a decrease in the blood supply to the penis and may also increase the drainage of blood from the erectile tissues. It is expected that this will produce a gradual reduction in the erection.
At the time of submission of the application for orphan designation no clinical trials in patients with low flow priapism had been initiated.
Etilefrine had not been marketed anywhere worldwide for low flow priapism 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 9 October 2002 recommending the granting of this designation.
- 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.
Key facts
- Active substance
- Etilefrine
- Intended use
- Treatment of low-flow priapism
- Orphan designation status
- Positive
- EU designation number
- EU/3/02/122
- Date of designation
- Sponsor
Laboratoires Serb
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: