EU/3/02/092

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Orphan designation

On 13 March 2002, orphan designation (EU/3/02/092) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for 4-(3,5-bis-(hydroxy-phenyl)-1,2,4)triazol-1-yl)-benzoic acid for the treatment of chronic iron overload requiring chelation therapy.

What is chronic iron overload requiring chelation therapy?

Chronic iron overload is a condition due to man’s inability to actively eliminate iron from the body. Chronic iron accumulation is mainly consecutive to either excess intestinal absorption (hemochromatosis) or excess administration through repetitive transfusions (iron contained in red blood cells). Repeated transfusions can be necessary in patients presenting chronic anaemias (e.g. thalassemia, sickle cell anaemia).

Chronic iron overload is a serious condition. Complications are related to iron deposits in tissues which can induce organ failure. This condition is life-threatening when the heart or the liver is affected.

What are the methods of treatment available?

Phlebotomy (blood removal by venipuncture) is the first-choice therapy for haemochromatosis, except when blood removal is impossible. For such patients, or for patients with transfusion-dependent anaemias, iron overload can be treated by administration of iron chelators.Two medicinal products which chelate iron had been authorised in the Community at the time of submission of the application for orphan designation. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that deferasirox might be of potential significant benefit for the treatment of chronic iron overload, particularly in terms of its pharmacological properties.

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

According to the information provided by the sponsor, chronic iron overload requiring chelation therapy was considered to affect about 102,000 persons in the European Union.

*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. 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?

Deferasirox is an iron chelator. Iron chelators are molecules binding to iron in the body, allowing it to then be eliminated through urinary or intestinal routes at a higher rate than the natural very low iron elimination rate (through shedding of skin and mucosal cells, menstruation or other blood loss).

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, clinical trials were ongoing in patients presenting iron overload after repeated transfusions for chronic anaemias.

Deferasirox had not been marketed anywhere worldwide or designated as an 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 23 January 2002 a positive opinion recommending the grant of the above mentioned designation.

Update: 4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid (Exjade) is authorised in the European Union as of 28 August 2006 for the treatment of chronic iron overload due to frequent blood transfusions ( >/= 7 ml/kg/month of packed red blood cells) in patients with beta thalassaemia major aged 6 years and older. Treatment of chronic iron overload due to blood transfusions when deferoxamine therapy is contraindicated or inadequate in the following patient groups:

  • in patients with other anaemias,
  • in patients aged 2 to 5 years,
  • in patients with beta thalassaemia major with iron overload due to infrequent blood transfusions (<7 ml/kg/month of packed red blood cells).
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 have been 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>4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid</p>
Active substance4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid
Medicine NameExjade
Disease/conditionTreatment of chronic iron overload requiring chelation therapy
Date of decision13/03/2002
OutcomePositive
Orphan decision numberEU/3/02/092

Review of designation

Sponsor’s contact details:

Novartis Europharm Limited
Wimblehurst Road
Horsham
West Sussex RH12 5AB
United Kingdom
Telephone: +44 (0)1403 272827
Telefax: +44(0)1403 323054

Patients' organisations:

For contact details of patients’ organisations whose activities are targeted at rare diseases see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe.
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.