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Orphan designation

On 27 July 2005, orphan designation (EU/3/05/300) was granted by the European Commission to Nerviano Medical Sciences Srl, Italy, for nemorubicin hydrochloride for the treatment of hepatocellular carcinoma.

What is hepatocellular carcinoma?

Tumours that begin in the liver are known as liver tumours. Liver tumours that have the potential to infiltrate healthy tissues and spread to other parts of the body are called hepatocellular carcinomas. The most common factors known to be associated with this disease are the viral infections causing liver inflammations (the so-called hepatitis B and hepatitis C) and an alcohol-induced liver disease (the socalled liver cirrhosis). Hepatocellular carcinoma is a life-threatening condition.

What are the methods of treatment available?

The choice of the treatment of hepatocellular cancer depends on several factors, mainly the stage of the disease. Treatments may include surgery, radiation therapy (using high-dose x-rays or other high-energy rays to kill cancer cells), chemotherapy (using drugs to kill cancer cells) or immunotherapy (treatment by stimulation of the body’s own defense system). At the time of submission of the application for orphan drug designation, several products were authorised for the condition in some countries of the Community.
Nemorubicin hydrochloride might be of potential significant benefit for the treatment of hepatocellular carcinoma, particularly in combination with currently authorised medicinal products, because it might improve the long-term outcome of the patients. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

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

According to the information provided by the sponsor, hepatocellular carcinoma was considered to affect about 46,000 persons in the European Union.

 *Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the EuropeanUnion (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. 

How is this medicinal product expected to act?

Nemorubicin hydrochloride is derived from doxorubicin by replacing a chemical group of its structure. Doxorubicin is a widely used drug to treat cancers and is also used in hepatocellular carcinoma. It is expected that nemorubicin hydrochloride, as doxorubicin, will also interfere with the production of genetic material in the cancer cells and will thereby, rapidly kill dividing cells such as cancer cells.

What is the stage of development of this medicinal product?

The effects of nemorubicin hydrochloride were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with hepatocellular carcinoma were ongoing. Nemorubicin hydrochloride was not authorised anywhere worldwide for hepatocellular carcinoma or designated as orphan medicinal product elsewhere for this condition, at the time of submission. According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 15 June 2005 a positive opinion recommending the grant of the above-mentioned designation.

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.

Key facts

Product details for <p>Nemorubicin hydrochloride</p>
Active substanceNemorubicin hydrochloride
Medicine Name
Disease/conditionTreatment of hepatocellular carcinoma
Date of decision27/07/2005
Orphan decision numberEU/3/05/300

Review of designation

Sponsor’s contact details:

Nerviano Medical Sciences Srl
Viale Pasteur 10
20014 Nerviano (Milano)
Telephone: +39 0331 58 11 11
Telefax: +39 0331 58 13 70

Patients’ association contact point:

The Association of European Cancer Leagues (ECL)
c/o Belgian Federation against Cancer
Chaussée de Louvain, 479
B-1030 Brussels
Telephone: +32 2 74 33 705
Telefax: +32 2 73 49 250

Cancer BACUP
3 Bath Place
Rivington Street
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
13 Av. de la Grande Armee
75116 Paris
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06