EU/3/20/2286: 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 4.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 239,000 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).

^*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of designation, the main treatments for soft tissue sarcoma were surgery and chemotherapy (medicines to treat cancer). Radiotherapy (treatment with radiation) was also used. Several medicines were authorised in the EU for the treatment of soft tissue sarcoma.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with soft tissue sarcoma because evidence from studies in patients suggest a positive response in patients for whom existing treatments have not worked.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine contains the patient’s own T cells (a type of white blood cell and part of the body’s natural defences) that have been modified genetically in the laboratory so that they can attach to cancer cells that produce a protein called MAGE-A4. 

When the medicine is given to the patient, the modified T cells are expected to attach to a part of MAGE-A4 which is present on the cancer cells and kill them, and to encourage other immune cells to attack the cancer. 

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 soft tissue sarcoma were ongoing.

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

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 23 April 2020, 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.