EU/3/16/1634 - orphan designation for treatment of soft tissue sarcoma

Glucopyranosyl lipid A stable emulsion and recombinant New York oesophageal squamous cell carcinoma-1 protein

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in October 2019 on request of the Sponsor. 

The sponsorship was transferred to Immune Design Ltd, United Kingdom, in August 2016.

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On 21 March 2016, orphan designation (EU/3/16/1634) was granted by the European Commission to Pharm Research Associates (UK) Limited, United Kingdom, for glucopyranosyl lipid A stable emulsion and recombinant New York oesophageal squamous cell carcinoma-1 protein (NY-ESO-1) for the treatment of soft tissue sarcoma.

What is 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.

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

At the time of designation, soft tissue sarcoma affected approximately 2.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 118,000 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 because early studies suggest that it may be of benefit in patients previously given other authorised cancer medicines. 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 works by stimulating the patient's immune system, the body's natural defences, so that it targets and destroys the cancer cells. It contains a protein called NY-ESO-1, which is found at high levels on cells in many soft tissue sarcomas. This is combined with a compound that helps to stimulate the immune system. When the medicine is given, the patient's immune system is expected to learn to treat NY-ESO-1 as 'foreign'. This is expected to stimulate an immune response against the cancer cells carrying NY-ESO-1 on their surface, resulting in the immune system attacking and destroying the cancer cells.

The medicine can be used together with another medicine (called 'Sindbis virus envelope pseudotyped lentiviral vector encoding New York oesophageal squamous cell carcinoma-1 protein') that helps the immune system to recognise the NY-ESO-1 protein as foreign and so stimulate the body to attack the cancer.

What is the stage of development of this medicine?

The effects of this 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 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 18 February 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/3/16/1634: Public summary of opinion on orphan designation: Glucopyranosyl lipid A stable emulsion and recombinant New York oesophageal squamous cell carcinoma-1 protein for the treatment of soft tissue sarcoma

Adopted Reference Number: EMA/COMP/159769/2016

Key facts

• Active substance:
  Glucopyranosyl lipid A stable emulsion and recombinant New York oesophageal squamous cell carcinoma-1 protein
• Intended use:
  Treatment of soft tissue sarcoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/16/1634
• Date of designation:
  21/03/2016
• Sponsor:

  Immune Design Ltd; 21 Holborn Viaduct; London EC1A 2DY; United Kingdom; Tel. [phone]; Contact us

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.