EU/3/11/871

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Orphan designation

On 21 June 2011, orphan designation (EU/3/11/871) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for salirasib for the treatment of pancreatic cancer.

The sponsorship was transferred to Kadmon International Ltd, United Kingdom, in November 2013.

What is pancreatic cancer?

Pancreatic cancer is cancer of the pancreas, a small organ that lies behind the stomach. The pancreas has two functions: to produce a juice that helps with the digestion of food, and to produce hormones such as insulin. Due to the absence of symptoms in the early stages of pancreatic cancer, the majority of patients are diagnosed when the cancer has spread locally or to other parts of the body.

Pancreatic cancer is a very severe and life-threatening disease that is associated with shortened life expectancy.

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

At the time of designation, pancreatic cancer affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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: 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. At the time of designation, this represented a population of 507,700,000 (Eurostat 2011).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for treating pancreatic cancer. The choice of treatment depended on several factors, including how advanced the disease is. Treatments included surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that salirasib might be of significant benefit for patients with pancreatic cancer because this medicine works in a different way to existing treatments and might be used in combination with existing treatments to improve the outcome of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Salirasib is expected to work by blocking ‘the Ras signalling pathway’. This is a mechanism within cells that helps them to grow and survive. However, in cancer cells it works abnormally, leading to the growth of the cancer. Salirasib is expected to attach to specific proteins on the membrane of cancer cells and thus detach proteins called Ras. By detaching Ras, the medicine is expected to block the signalling pathway, thereby slowing down the growth of the cancer.

What is the stage of development of this medicine?

The effects of salirasib have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with salirasib in patients with pancreatic cancer were ongoing.

At the time of submission, salirasib was not authorised anywhere in the EU for the treatment of pancreatic cancer. Orphan designation of salirasib had been granted in the United States for the treatment of pancreatic cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 March 2011 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.

Name Language First published Last updated
EU/3/11/871: Public summary of opinion on orphan designation: Salirasib for the treatment of pancreatic cancer (English only) 12/07/2011 14/03/2013

Key facts

Product details for <p>Salirasib</p>
Active substanceSalirasib
Medicine Name
Disease/conditionTreatment of pancreatic cancer
Date of decision21/06/2011
OutcomePositive
Orphan decision numberEU/3/11/871

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

Kadmon International Ltd
4th Floor
Haines House
21 John Street
London
WC1N 2BP
United Kingdom
Tel. +44 (0)20 7692 1917
Fax: +44 (0)20 7692 1918

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.