EU/3/06/356

  • Email
  • Help

Orphan designation

On 16 February 2006, orphan designation (EU/3/06/356) was granted by the European Commission to Prodimed S.A., Spain , for 4-amino-5-oxo-4 (pyridinium-1-ylmethyl) proline for the treatment of renal cell carcinoma.

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 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 the tubules in the kidney. Renal cell carcinoma accounts for approximately 85% of all kidney cancers. Signs of 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. 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 approximately 3.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 161,000 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 based on data from the European  Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

There are treatments for most patients with renal cell carcinoma. 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-alpha. Other anticancer agents had also been authorised in the Community for treatment of renal cell carcinoma at the time of submission of the application for orphan designation.
Satisfactory argumentation has been submitted by the sponsor to justify the assumption that 4-amino-5-oxo-4 (pyridinium-1-ylmethyl) proline might be of potential significant benefit for the treatment of renal cell carcinoma because it might act in a different way than other available medicines, and thereby it might improve the long-term outcome of the patients. 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?

At the time of the submission of the orphan drug designation application, it was not completely understood how 4-amino-5-oxo-4 (pyridinium-1-ylmethyl) proline works in the human body. It is thought that 4-amino-5-oxo-4 (pyridinium-1-ylmethyl) proline may help the body’s immune system to kill the cancer cells.

What is the stage of development of this medicine?

The effects of 4-amino-5-oxo-4 (pyridinium-1-ylmethyl) proline were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with renal cell carcinoma were initiated.

The medicinal product was not authorised anywhere worldwide 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 11 January 2006 recommending the granting of this designation.

Opinions on orphan medicinal products designations are based on the following cumulative 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>4-Amino-5-oxo-4 (pyridinium-1-ylmethyl) proline</p>
Active substance4-Amino-5-oxo-4 (pyridinium-1-ylmethyl) proline
Medicine Name
Disease/conditionTreatment of renal cell carcinoma
Date of decision16/02/2006
OutcomePositive
Orphan decision numberEU/3/06/356

Review of designation

Sponsor’s contact details:

Prodimed S.A.
C/Guzman el Bueno, 74. 1°
28015 MadridSpain
Telephone: +34 91 535 16 72
Telefax: +34 91 456 07 23
E-mail: info@prodimed.es

Patients’ associations contact points:

Kidney Cancer UK
11 Hathaway Road
Tile Hill Village
CV4 9HW
Coventry
Telephone: +44 24 76 47 49 93
E-mail: kidneycanceruk@hotmail.com

Cancer BACUP
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
Telephone: +44 20 76 96 90 03 / 0808 800 1234 (freephone for UK)
Telefax: +44 20 76 96 90 02

Ligue Nationale contre le Cancer (LNCC)
12, rue Corvisart
75013 Paris
France
Tel : +33 1 53 55 24 00
Telefax : +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net