EU/3/16/1694: Orphan designation for the treatment of soft tissue sarcoma

Soft tissue sarcoma is a 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 approximately 2.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 144,000 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 513,700,000 (Eurostat 2016).

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 cancer 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 medicine might be of significant benefit for patients with soft tissue sarcoma. The response rate in early studies with the medicine compared well with response rates seen in medical literature with other authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is made up of T cells taken from the patient. T cells are cells of the immune system (the body's natural defences) that kill infected or abnormal cells. To make this medicine, the T cells are modified by a virus known as a 'lentivirus'. The virus carries a gene that enables the T cells to produce a receptor that recognises the protein NY-ESO-1 as foreign. NY-ESO-1 is found at high levels on cells in many soft tissue sarcomas. When these modified T cells are given back to the patient, they are expected to recognise and kill cancer cells producing NY-ESO-1, thus reducing the growth and spread of the tumour.

The virus has been designed so that it does not enter other types of cells and cannot reproduce itself and cause disease in the body.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with various cancers were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for soft tissue sarcoma. Orphan designation has been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 June 2016 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.