EU/3/15/1447:

Soft tissue sarcoma is a type of cancer that affects the soft, supportive tissues of the body. It can occur in muscles, blood vessels, fat tissue or in other tissues that support, surround and protect organs. Patients with soft tissue sarcoma do not usually have symptoms in the early stages of the disease. First symptoms appear when the tumour grows large enough to cause swelling and pain.

Soft tissue sarcoma is a long-term debilitating and life-threatening disease, particularly when the cancer has spread to other parts of the body.

At the time of designation, soft tissue sarcoma affected not more than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 154,000 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 on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).

At the time of designation, the main treatment for early-stage soft tissue sarcoma was surgery. For large sarcomas, surgery was usually followed by radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer) to kill any cancerous cells that were left behind. Several medicines were authorised in the EU for the treatment of soft tissue sarcoma including doxorubicin.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with soft tissue sarcoma because early studies indicate that it might improve the outcome of patients when combined with doxorubicin. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Olaratumab is a monoclonal antibody, a type of protein that has been designed to recognise and attach to a protein called platelet-derived growth factor receptor alpha (PDGFRα). This protein is often found on the surface of cells where it is thought to play a role in regulating cell multiplication. In cancers such as soft tissue sarcoma these proteins are present in high levels or overactive, causing cells to become cancerous. When olaratumab attaches to PDGFRα on sarcoma cells, it is expected to block its activity, thereby slowing down the growth of the cancer.

The effects of olaratumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with olaratumab in patients with soft tissue sarcoma were ongoing.

At the time of submission, olaratumab was not authorised anywhere in the EU for soft tissue sarcoma. Orphan designation of olaratumab had been granted in the United States of America for the treatment of soft tissue sarcoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 2015 recommending the granting of this designation.

• 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.