EU/3/02/115: Orphan designation for the treatment of uraemic pruritus

Uremic pruritus is a severe and persisting form of itching which occurs in some patients whose kidneys are not functioning properly. Because of kidney failure, these patients have to undergo a process of filtering of accumulated waste products from the blood (haemodialysis) or the peritoneum, a membrane which lines the abdomen (peritoneal dialysis). Uremic pruritus can be severe and impair the patients' quality of life.

At the time of designation, uremic pruritus affected approximately 3.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 132,000 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: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 377,000,000 (Eurostat 2001) and may differ from the true number of patients affected by the condition.

There were no authorised products for the treatment in the Community. Products which are frequently used in the common forms of itching have only limited efficacy in many patients.

It seems that the itching is linked to an imbalance in opium-similar substances that are produced naturally in the brain. (-)-17-(cyclopropylmethyl)-3,14 ß-dihydroxy-4,5 α-epoxy-6ß-[N-methyl-trans-3-(3-furyl) acrylamido] morphinan hydrochloride (intravenous use) is expected to activate a receptor
(called kappa-receptor). Stimulation of this receptor of the central nervous system would alleviate the uremic pruritus.

At the time of submission of the application for orphan designation, clinical trials in patients with uremic pruritus were ongoing.

(-)-17-(cyclopropylmethyl)-3,14 ß-dihydroxy-4,5 α-epoxy-6ß-[N-methyl-trans-3-(3-furyl) acrylamido] morphinan hydrochloride (intravenous use) had not been marketed anywhere worldwide for uremic pruritus 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 1 August 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