EU/3/05/336

On 23 December 2005, orphan designation (EU/3/05/336) was granted by the European Commission to Nerviano Medical Sciences Srl, Italy, for brostallicin for the treatment of soft tissue sarcoma.
The sponsorship was transferred to AAIPharma S.A., France, in February 2009.

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, which tend to behave differently. 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 2.4 in 10,000 people in the European Union (EU)*. This is equivalent to a total of 110,000 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?

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.
Brostallicin could be of potential significant benefit for the treatment of soft tissue sarcomas, mainly because 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?

Brostallicin is expected to bind to the fundamental genetic material (DNA, the cellular structure which carries the genetic information). Since the DNA is necessary for the division and survival of both normal and abnormal (i.e. tumour) cells, brostallicin might slow down or kill cancer cells which are actively dividing cells.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with soft tissue sarcoma were ongoing.
The medicinal product 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 10 November 2005 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.