Overview

This medicine is now known as nalfurafine.

On 11 September 2002, orphan designation (EU/3/02/115) was granted by the European Commission to Toray Europe Limited, United Kingdom, for (-)-17-(cyclopropylmethyl)-3,14 ß-dihydroxy-4,5 α-epoxy-6ß-[N-methyl-trans-3-(3-furyl) acrylamido] morphinan hydrochloride (intravenous use) for the treatment of uremic pruritus.

Toray Europe Limited changed name to Toray International U.K. Limited in January 2008.

The sponsorship was transferred to Toray International Europe GmbH, Germany, in February 2017.

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 ?-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 ?-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

EU/3/02/115: Public summary of positive opinion for orphan designation of (-)-17-(cyclopropylmethyl)-3,14 àŸ-dihydroxy-4,5 α-epoxy-6àŸ-[N-methyl-trans-3-(3-furyl) acrylamido] morphinan hydrochloride (intravenous use) fo...

AdoptedReference Number: EMA/COMP/78338/2004 Rev.4

English (EN) (102.02 KB - PDF)

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Key facts

Intended use
Treatment of uraemic pruritus
Orphan designation status
Positive
EU designation number
EU/3/02/115
Date of designation
Sponsor

Toray International Europe GmbH
Hugenottenallee 175
63263 Neu-Isenburg
Germany
Tel. +49 (0) 6102 79990
E-mail:  medical@toray-intl.de

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

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:

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