EU/3/07/449 - orphan designation for treatment of renal-cell carcinoma

everolimus

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2011 on request of the sponsor.

On 5 June 2007, orphan designation (EU/3/07/449) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for everolimus for the treatment of renal cell carcinoma.

Everolimus has been authorised in the EU as Afinitor since 3 August 2009.

What is renal cell carcinoma?

Renal cell carcinoma (also called cancer of the kidney or renal adenocarcinoma) is a disease in which cancer (malignant) cells are found in certain tissues of the kidney. Inside each kidney there are tiny tubules that filter and clean the blood, taking out waste products, and making urine. Renal cell carcinoma is a cancer of the lining of these tubules in the kidney. Renal cell carcinoma accounts for approximately 85% of all kidney cancers. Signs of this cancer are difficult to detect in early stages of the disease, and about half of the patients are diagnosed when the disease has spread around the kidney or to distant parts of the body. Surgery is a common treatment of renal cell cancer, and allows taking out the cancer in an operation, although the cancer may appear again. Renal cell carcinoma is life-threatening.

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

At the time of designation, renal cell carcinoma affected less than 4.2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 210,000 people*, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP).

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*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 500,300,000 (Eurostat 2007).

What treatments are available?

There are treatments for most patients with renal cell cancer. These may include surgery (taking out the cancer in an operation), chemotherapy (using drugs to kill cancer cells), radiation therapy (using high-dose x-rays or other high-energy rays to kill cancer cells), hormone therapy (using hormones to stop cancer cells from growing), and biological therapy (using the body's immune system to fight cancer). The primary therapies for advanced cancer are biologic agents, such as interleukin-2 and interferon-?. Recently, other anticancer agents (sorafenib and sunitinib) aimed at stopping cancer cells growth have also been authorised in the Community for the treatment of renal cell carcinoma.

The sponsor submitted satisfactory argumentation to justify the assumption that everolimus might be of potential significant benefit for the treatment of renal cell carcinoma. This could represent an additional treatment option for patients with renal cell carcinoma. The assumption will have to be confirmed at the time of marketing authorisation; this will be necessary to maintain the orphan status.

How is this medicine expected to work?

Enzymes are proteins produced by the human body that speed up the conversion of certain chemical substances of the body into other substances. Everolimus blocks (inhibits) a particular enzyme, called serine/threonine kinase. This enzyme plays a role in a number of molecular reactions, which control the growth and the division of the cells. In cancer cells, the function of this enzyme is disturbed, causing uncontrolled growth and multiplication of the cancer cells. Everolimus might, by inhibition of this enzyme activity, help in slowing down or stopping the further growth of the cancer cells.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with renal cell carcinoma were ongoing.

Everolimus was not authorised anywhere in the world for renal cell carcinoma or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 April 2007 recommending the granting of this designation.

Update: Everolimus (Afinitor) has been authorised in the EU since 3 August 2009 for the treatment of patients with advanced renal cell carcinoma, whose disease has progressed on or after treatment with VEGF-targeted therapy.

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.

EU/3/07/449: Public summary of positive opinion for orphan designation of everolimus for the treatment of renal cell carcinoma

Adopted Reference Number: EMEA/COMP/202020/2007 Rev. 3

Key facts

• Active substance:
  everolimus
• Medicine name:
  Afinitor
• Intended use:
  Treatment of renal-cell carcinoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/07/449
• Date of designation:
  05/06/2007
• Sponsor:

  Novartis Europharm Limited

Review of designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products on July 2011 on request of the sponsor.