EU/3/12/1071

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Orphan designation

On 8 November 2012, orphan designation (EU/3/12/1071) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for canakinumab for the treatment of tumour-necrosis-factor-receptor-associated periodic syndrome.

What is tumour-necrosis-factor-receptor-associated periodic syndrome?

Tumour-necrosis-factor-receptor-associated periodic syndrome (TRAPS) is an inflammatory disease in which patients have recurrent episodes of fever, rash and pain in various parts of the body, including the eyes, joints, muscles and the abdomen. These symptoms are caused by excessive inflammation as a result of a mutation in the gene for proteins called ‘tumour-necrosis-factor (TNF) receptors’, which play an important role in the inflammatory process.

TRAPS is a long-term disease that is debilitating due to its recurrent symptoms and the development of kidney complications.

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

At the time of designation, TRAPS affected approximately 0.01 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 500 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: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

What treatments are available?

At the time of the orphan designation, no satisfactory treatments for TRAPS were authorised in the European Union (EU). Anti-inflammatory medicines such as non-selective non-steroidal anti-inflammatory drugs (NSAIDs) and glucocorticosteroids were used to manage the symptoms of the disease.

How is this medicine expected to work?

Patients with TRAPS have excessive activity of inflammatory proteins, including a protein called ‘interleukin-1 beta’, which is involved in causing the inflammation in these patients. Canakinumab is a monoclonal antibody (a type of protein) designed to attach to interleukin-1 beta and block its activity, thereby reducing the inflammation associated with the disease.

Canakinumab is authorised in the EU for the treatment of another inflammatory disease, cryopyrin-associated periodic syndrome (CAPS), in which it also targets interleukin-1 beta.

What is the stage of development of this medicine?

The effects of canakinumab have been evaluated in experimental models.

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

At the time of submission, canakinumab was not authorised anywhere in the EU for TRAPS. Orphan designation has been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 October 2012 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • 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.

Key facts

Product details for <p>Canakinumab</p>
Active substanceCanakinumab
Medicine Name
Disease/conditionTreatment of tumour-necrosis-factor-receptor-associated periodic syndrome
Date of decision08/11/2012
OutcomePositive
Orphan decision numberEU/3/12/1071

Review of designation

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

Sponsor’s contact details:

Novartis Europharm Limited
Wimblehurst Road
Horsham
West Sussex RH12 5AB
United Kingdom
Tel. +44 (0)1403 32 42 60
Fax: +44 (0)1403 32 30 54
E-mail: nicki.hiller@novartis.com

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.