EU/3/09/687 - orphan designation for treatment of primary peritoneal cancer

patupilone

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in January 2011 on request of the Sponsor.

On 5 November 2009, orphan designation (EU/3/09/687) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for patupilone for the treatment of primary peritoneal cancer.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is primary peritoneal cancer?

Primary peritoneal cancer is a rare cancer that starts in the peritoneum. This is the membrane that forms the lining of the abdominal (tummy) cavity. The cells that form the peritoneum ('epithelial cells') are the same as those lining the ovaries, where most of the ovarian cancers start. Therefore, this type of cancer shares some characteristics with ovarian cancer. Primary peritoneal cancer mainly affects women aged between 40 and 60 years.

Primary peritoneal cancer is a severe and life-threatening disease that leads to poor long-term survival.

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

At the time of designation, primary peritoneal cancer affected approximately 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 15,000 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 the 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 504,800,000 (Eurostat 2009).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of primary peritoneal cancer. Because of the similarities between ovarian and primary peritoneal cancer cells, the treatments authorised for ovarian cancer were used also for primary peritoneal cancer.

How is this medicine expected to work?

Patupilone is a substance extracted from bacteria. It is a cytotoxic (a substance that kills cells that are dividing, such as cancer cells). It is expected to work by blocking the cell's ability to break down the 'skeleton' that allows cells to divide and multiply. With the skeleton still in place the cells cannot divide, and they eventually die.

What is the stage of development of this medicine?

The effects of patupilone have been evaluated in experimental models.

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

At the time of submission, patupilone was not authorised anywhere in the EU for primary peritoneal cancer or designated as orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 2 September 2009 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 Community) 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.

EU/3/09/687: Public summary of positive opinion for orphan designation of patupilone for the treatment of primary peritoneal cancer

Adopted Reference Number: EMEA/COMP/582084/2009 Rev. 2

Key facts

• Active substance:
  patupilone
• Intended use:
  Treatment of primary peritoneal cancer
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/09/687
• Date of designation:
  05/11/2009
• Sponsor:

  Novartis Europharm Limited