EU/3/04/229

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Orphan designation

On 21 October 2004, orphan designation (EU/3/04/229) was granted by the European Commission to BioAlliance Pharma, France, for doxorubicin polyisohexylcyanoacrylate nanoparticles 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 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 so-called liver cirrhosis). Hepatocellular carcinoma is a life-threatening condition.

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

At the time of designation, hepatocellular carcinoma affected approximately 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 46,000 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).

*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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

No medicinal products for the treatment of the condition had been authorised in the European Union at the time of submission of the application for orphan drug designation.

How is this medicine expected to work?

Doxorubicin is a drug to treat tumours. Doxorubicin interferes with the production of genetic material and can kill rapidly dividing cells such as tumour cells. In the designated product, doxorubicin is entrapped in nanoparticles of polyisohexylcyanoacrylate. Once administered to the patient, the nanoparticles are adsorbed to the surface of tumour cells and doxorubicin entrapped in these particles is subsequently released on the cancer cell. The nanoparticles degrade and release also polyisohexylcyanoacrylate, which may contribute to delivering doxorubicin into the cancer cells. This may increase the presence of the drug in the tumour cells and may enhance its activity.

What is the stage of development of this medicine?

The effects of doxorubicin polyisohexylcyanoacrylate nanoparticles 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.
Doxorubicin polyisohexylcyanoacrylate nanoparticles was not marketed 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 9 September 2004 a positive opinion recommending the grant of the above-mentioned 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.

Key facts

Product details for <p>Doxorubicine polyisohexylcyanoacrylate nanoparticles</p>
Active substanceDoxorubicine polyisohexylcyanoacrylate nanoparticles
Medicine Name
Disease/conditionTreatment of hepatocellular carcinoma
Date of decision21/10/2004
OutcomePositive
Orphan decision numberEU/3/04/229

Review of designation

Sponsor’s contact details:

BioAlliance Pharma
49 Boulevard du Général Martial Valin
F-75015 Paris
France
Telephone: +33 1 45 58 76 00
Telefax: +33 1 45 58 08 81
E-mail: bioalliance@bioalliancepharma.com

Patients associations’ contact points:

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
France
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06
E-mail: ligue@ligue-cancer.net

British Liver Trust
Portman House 
44 High Street 
Ringwood BH24 1AG
United Kingdom 
Telephone: +44 14 25 46 30 80 
E-mail info@britishlivertrust.org.uk

CancerBACUP
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
Telephone: +44 20 76 96 90 03 (from the UK 0808 800 1234)
Telefax: +44 20 76 96 90 02