EU/3/14/1258: Orphan designation for the treatment of soft tissue sarcoma

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 2 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 103,000 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.
At the time of designation, this represented a population of 512,900,000 (Eurostat 2014).

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.

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 in patients showed better response rates than existing treatments. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Doxorubicin has been available as an anticancer medicine since the 1960s. It is a cytotoxic (cell-killing) medicine that belongs to the group 'anthracyclines'. It works by interfering with the DNA within cells, preventing them from making more copies of DNA and making proteins. This means that cancer cells cannot divide and eventually die.

In this medicine, doxorubicin is linked to a small molecule that attaches to albumin, a blood protein which is known to accumulate in tumours such as soft tissue sarcoma. Albumin is therefore expected to act as a carrier and transport the medicine through the blood vessels directly to the tumour environment where doxorubicin is expected to be released. Once released, doxorubicin is expected to act to kill the cancer cells, thus improving the symptoms or the disease.

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 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 of 16 December 1999, the COMP adopted a positive opinion on 6 February 2014 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.