EU/3/05/324

  • Email
  • Help

Orphan designation

On 28 October 2005, orphan designation (EU/3/05/324) was granted by the European Commission to ACE Pharmaceuticals BV, The Netherlands, for levamisol hydrochloride for the treatment of nephrotic syndrome.

What is nephrotic syndrome?

Nephrotic syndrome is a type of kidney disorder that is characterised by oedema (presence of abnormal large amounts of fluid in the body) and some abnormal amounts of certain substances such as “low serum albumin” (a major type of protein in the blood) and a large amount of protein in the urine. Other typical signs or symptoms of the disease apart from oedema, are weight gain, high blood pressure and an uncontrolled lack or loss of appetite. Nephrotic syndrome might occur without apparent causes (especially in children) or as a result of a number of illnesses that can damage the kidney.
Nephrotic syndrome is a chronically debilitating and life-threatening condition.

What are the methods of treatment available?

Medicinal products, such as corticosteroids, were authorised for the condition within the Community at the time of submission of the application for orphan drug designation. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that the medicinal product might be of potential significant benefit for the treatment of nephrotic syndrome, particularly in terms of its corticosteroid-sparing effect. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

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

According to the information provided by the sponsor, nephrotic syndrome was considered to affect about 46,000 persons in the European Union.

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

Nephrotic syndrome is considered as an immunologic disease (involving a reaction by which the body harms itself). Levamisol hydrochloride is proposed to act by changing the body’s immune response against the disease. As a result the need for treatment with immunosuppressants, e.g. corticosteroids, could be reduced.

What is the stage of development of this medicinal product?

The effects of levamisole hydrochloride were evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials in patients with nephrotic syndrome were initiated.
The medicinal product was not authorised anywhere worldwide for nephrotic syndrome or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 9 September 2005 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition,
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>Levamisol hydrochloride</p>
Active substanceLevamisol hydrochloride
Medicine Name
Disease/conditionTreatment of nephrotic syndrome
Date of decision28/10/2005
OutcomePositive
Orphan decision numberEU/3/05/324

Review of designation

Sponsor’s contact details:

ACE Pharmaceuticals BV
Schepenveld 41
3891 ZK Zeewolde
The Netherlands
Telephone: +31 36 522 72 01
Telefax: +31 36 522 9096
E-mail: ace@ace-pharm.nl

Patients’ associations contact points:

Association des Malades d'un Syndrome Néphrotique (AMSN)
11, avenue de la Roseraie
91440
Bures Sur Yvette
France
Telephone: +33 1 69 86 12 06
E-mail: syndrome.nephrotique@laposte.net

The British Kidney Patient Association
Bordon. Hants
GU35 9JZ
United Kingdom
Telephone: +44 14 20 47 20 21 / 2
Telefax: +44 14 20 47 58 31

AIRG-E : Asociación para la información e investigación sobre enfermedades renales genéticas
Fundació Puigvert
c/ Cartagena, 340-350
08025 Barcelona
Spain
Telephone: +34 65 94 63 198
Telefax: +34 93 41 69 730
E-mail: airg@menta.net