EU/3/07/502

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Orphan designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in January 2012 on request of the Sponsor.

On 6 December 2007, orphan designation (EU/3/07/502) was granted by the European Commission to Kendle International, UK, for picoplatin for the treatment of small-cell lung cancer.

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

What is small-cell lung cancer?

Small-cell lung cancer is a disease in which cancer (malignant) cells develop in the lungs. This type of lung cancer accounts only for about 20% of all lung cancer cases. Small-cell cancer usually develops in the central part of the lungs, and the cancer cells are typically small compared with the other types of lung cancer. Small-cell cancer is difficult to detect in early stages of the disease, and the majority of the patients are diagnosed when the disease has spread and cannot be surgically removed. Small-cell lung cancer is a life-threatening disease.

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

At the time of designation, small-cell lung cancer affected less than 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 75,000 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 500,300,000 (Eurostat 2007).

What treatments are available?

The main treatment of small-cell lung cancer consists of chemotherapy (i.e drugs that kill cancer cells) and radiotherapy (i.e using high-dose x-rays or other high-energy rays to kill cancer cells).

Several medicinal products were authorised in the European Community, for the treatment of small-cell lung cancer, at the time of submission of the application for orphan designation. Picoplatin might be of potential significant benefit for the treatment of small-cell lung cancer, because it might improve the long-term outcome. This 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?

Platinum-containing products are already authorized for the treatment of small-cell lung cancer. Picoplatin is a platinum-derivative that is able to bind to the genetic material of cells (DNA) and to interfere with its normal function, leading to lack of DNA replication, absence of protein production from DNA and, ultimately, cell death. Picoplatin has been designed to overcome two of the common problems observed during treatment with other platinum compounds; namely, the onset of resistance to drug treatment (tumour growth continues or re-starts) and the triggering of important adverse reactions. These characteristics of picoplatin may be of potential significant benefit over the existing authorized medicinal products. This assumption will have to be confirmed at the time of marketing authorisation, and this will be necessary to maintain the orphan status.

What is the stage of development of this medicine?

The effects of picoplatin were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with small-cell lung cancer were ongoing.

Picoplatin was not authorised anywhere in the world for the treatment of small-cell lung cancer, at the time of submission. Orphan designation was granted in the United States on 2 November 2005 to cis-Amminedichloro(2-methylpyridine)platinum(II) (the chemical name of picoplatin) for the treatment of small cell lung cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 October 2007 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>Picoplatin</p>
Active substancePicoplatin
Medicine Name
Disease/conditionTreatment of small cell lung cancer
Date of decision05/12/2007
OutcomeWithdrawn
Orphan decision numberEU/3/07/502

Review of designation

Sponsor’s contact details:

Kendle International Ltd
River View
The Meadows Business Park
Station Approach, Camberley
Surrey GU17 9AB
United Kingdom
Telephone: +44 1276 481 000
Telefax: +44 1276 35743
E-mail: info.ely@kendle.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.