EU/3/16/1739 - orphan designation for treatment of soft tissue sarcoma

A non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format

Overview

On 14 October 2016, orphan designation (EU/3/16/1739) was granted by the European Commission to Philogen S.p.A., Italy, for a non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format (also known as L19TNF) for treatment of soft tissue sarcoma.

What is soft tissue sarcoma?

Soft tissue sarcoma is a cancer that affects the soft 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.

What is the estimated number of patients affected by the condition?

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).

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* 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).

What treatments are available?

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 this medicine might be of significant benefit for patients with soft tissue sarcoma, with early data showing improved progression-free survival (how long patients lived without their disease getting worse) in patients who had received previous treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine is made of two joined proteins, an antibody and tumour necrosis factor (TNF). The antibody is a protein designed to attach to blood vessels that supply the tumours, while TNF is a natural protein known to attack and kill cells. By having the antibody and TNF joined together, the medicine is expected to target and kill soft tissue sarcoma cells, helping to slow down progression of the cancer.

What is the stage of development of this medicine?

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 including 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 or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 September 2016 recommending the granting of this 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 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.

EU/3/16/1739: Public summary of positive opinion for orphan designation of a non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragm...

Adopted Reference Number: EMA/628407/2016

Key facts

• Active substance:
  A non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format
• Intended use:
  Treatment of soft tissue sarcoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1739
• Date of designation:
  20/09/2016
• Sponsor:

  Philogen S.p.A.; La Lizza 7; 53100 Siena; Italy; Tel. [phone]; Fax [phone]; E-mail: [e-mail]