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Orphan designation

On 27 October 2006, orphan designation (EU/3/06/410) was granted by the European Commission to GP-Pharm S.A., Spain, for doxorubicin hydrochloride (liposomal) for the treatment of soft tissue sarcoma.

What is soft tissue sarcoma?

Soft tissue sarcomas are cancers of the supporting tissues of the body. They can occur in muscle, fat, blood vessels or in any of the other tissues that support, surround and protect the organs of the body. There are many types of soft tissue sarcoma. The cause of soft tissue sarcoma is largely unknown. Soft tissue sarcoma is a serious condition, potentially debilitating and life-threatening.

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

At the time of designation soft tissue sarcoma affected approximately 2.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 115,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

Surgery is currently the main choice of therapy for early stage soft tissue sarcomas. For larger sarcomas, and where it is thought there is a possibility of cancer cells being left behind, after a surgical intervention, radiotherapy (using high-dose x-rays or other high-energy rays to kill cancer cells) and chemotherapy (using drugs to kill cancer cells) are usually used. Several products for treatment of soft tissue sarcoma were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that doxorubicin hydrochloride (liposomal) might be of potential significant benefit for the treatment of soft tissue sarcoma mainly 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.

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 located in the wall of a liposome (a microscopic particle made out of the same material as a cell membrane that can be filled with drugs). This allows the liposomes to circulate for prolonged periods in the blood stream and thus might maintain a longer effect.

What is the stage of development of this medicine?

The effects of doxorubicin hydrochloride (liposomal) were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with soft tissue sarcoma were initiated.

Doxorubicin hydrochloride (liposomal) was not authorised anywhere worldwide for soft tissue sarcoma 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 6 September 2006 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>Doxorubicin hydrochloride (liposomal)</p>
Active substanceDoxorubicin hydrochloride (liposomal)
Medicine Name
Disease/conditionTreatment of soft tissue sarcoma
Date of decision27/10/2006
Orphan decision numberEU/3/06/410

Review of designation

Sponsor’s contact details:

GP-Pharm S.A.
Pol. Ind. Els Vinyets els Fogars 2
Carretera Comarcal 244, km 22
08777 St Quintí de Mediona
Telephone: +34 93 819 22 00
Telefax: +34 93 638 93 93

Patients’ associations contact points:

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10

Deutsche Krebshilfe e. V.
Buschstr. 32
53113 Bonn
Telephone: +49 2 28 729 900
Telefax: +49 2 287 299 011

Macmillan Cancer Support (merged with CancerBACUP)
3 Bath Place
Rivington Street
London EC2A 3JR
United Kingdom
Telephone: +44 20 7696 9003
Switchboard open during office hours, Mon–Fri, 09:00 – 12:00 and 14:00 –16:45.
Telefax: +44 20 7696 9002