EU/3/05/302

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Orphan designation

On 26 August 2005, orphan designation (EU/3/05/302) was granted by the European Commission to Xechem UK Ltd., United Kingdom, for extract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineense seed and Caryophylli flower for the treatment of sickle cell disease.

What is sickle cell disease?

Sickle cell disease is a group of inherited diseases caused by a mutation in a blood protein called haemoglobin, leading to a so-called “hemoglobin S”. The mutation leads to changes in the shape and behaviour of red blood cells. They become hard, sticky and shaped in the form of a sickle (the sharp hooks used to cut the wheat). This causes small blood clots that block blood flow, and cause repeated painful episodes, called crisis. In the long run, this causes severe damage to the organs in the body. The clinical picture of patients with sickle cell disease can be remarkably variable. Some patients remain without much of complaints, while others suffer repeated crises requiring admission to hospital from early childhood.

What are the methods of treatment available?

No medicines exist that have been authorised for the treatment of sickle cell disease. Oxygen, blood transfusions, and narcotics for pain, help to improve the symptoms of the crisis. Bone marrow transplantation can provide a cure, but it is known to be effective and safe only in selected children. 

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

According to the information provided by the sponsor, sickle cell disease was considered to affect less than 78,000 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?

Red blood cell sickling involves the polymerisation (molecules joining together into a chain) of hemoglobin S. Extract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineense seed and Caryophylli flower was shown to block (inhibit) the polymerisation of this hemoglobin S and might therefore reduce the number of sickle cells in the blood. This might help to reduce the severity of the disease.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, clinical trials in patients with sickle cell disease were ongoing. 
Extract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineense seed and Caryophylli flower was not marketed anywhere worldwide for treatment of sickle cell disease, at the time of submission. Orphan designation of the medicinal product (as “Niprisan”) was granted in USA for treatment of sickle cell disease. According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 13 July 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>Extract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineense seed and Caryophylli flower</p>
Active substanceExtract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineense seed and Caryophylli flower
Medicine Name
Disease/conditionTreatment of sickle cell disease
Date of decision26/08/2005
OutcomePositive
Orphan decision numberEU/3/05/302

Review of designation

Sponsor’s contact details:

Xechem UK Ltd
The White House
2 Meadrow
Godalming
Surrey GU7 3HN
United Kingdom
Telephone: +44 20 86 41 30 35
Telefax: +44 87 04 28 25 52
E-mail: martin.biggs@mba-gbm.com

Patients’ associations contact points:

Sickle Cell Society
54 Station Road
London
NW10 4UA
United Kingdom
Telephone: +44 (0) 20 8961 7795
Telefax : +44 (0) 20 8961 8346
E-mail: info@sicklecellsociety.org

APIPD : Association pour l'Information et la Prévention de la Drépanocytose
7 ter Rue Edouard Vaillant
93400 Saint-Ouen
France
Telephone :+33 1 40 10 02 49
Telefax : +33 1 40 10 96 38
E-mail: jenny.hippocrate@free.fr

Associazione Talassemici e Drepanocitici Lombardi (A.T.D.L)
c/o A.V.I.S. Comunale
L.go Volontari del Sangue, 1
20133 Milano
Italy
Telephone: +39 02 70 63 87 96
Telefax: +39 1 78 22 89 515
E-mail: info@atdl.it