EU/3/16/1655

On 28 April 2016, orphan designation (EU/3/16/1655) was granted by the European Commission to SELLAS Life Sciences Group UK, Limited, United Kingdom, for Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-Leu-Val-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-Met-Thr-Lys-Leu and Pro-Gly-Cys-Asn-Lys-Arg-Tyr-Phe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-Arg-Lys-His-Thr-Gly for the treatment of malignant mesothelioma.

What is malignant mesothelioma?

Malignant mesothelioma is a cancer that affects the mesothelial cells (found on the inner linings of the organs), mainly in the pleura (the lining of the lungs) and in the peritoneum (the lining of the abdominal cavity). It is usually caused by exposure to asbestos. Mesothelioma of the pleura causes difficulty breathing and chest pain, and mesothelioma of the peritoneum causes ascites (a build-up of fluid in the abdomen) and abdominal pain.

Malignant mesothelioma is life-threatening because it may lead to bowel obstruction, heart or breathing problems and lung infections. Patients have very poor survival, only living for a year, on average, after diagnosis.

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

At the time of designation, malignant mesothelioma affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 treatments for malignant mesothelioma were surgery and chemotherapy (medicines to treat cancer) with or without radiotherapy (treatment with radiation). If the disease was too advanced for surgery, chemotherapy alone was used. Only one medicine, pemetrexed, was authorised throughout the EU for the treatment of malignant pleural mesothelioma. A second medicine, raltitrexed, was authorised for the treatment of malignant pleural mesothelioma in a small number of European countries.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with malignant mesothelioma because early clinical studies showed that it might improve the overall survival of patients who have completed previous treatments, including surgery. 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?

This medicine works by stimulating the patient’s immune system, the body’s natural defences, so that it targets and destroys the cancer cells. The medicine contains four amino acid chains and an adjuvant (a compound that enhances the immune response). The amino acid chains are similar to parts of a protein called Wilms’ tumour 1 present in many cancer cells including mesothelioma cells. When the medicine is given, the patient’s immune system is expected to learn to treat Wilms’ tumour 1 as ‘foreign’. This is expected to stimulate an immune response against the mesothelioma cells producing Wilms’ tumour 1, resulting in the immune system attacking and destroying these cancer cells.

What is the stage of development of this medicine?

The sponsor has provided data in experimental models from the published literature to support its application for orphan designation.

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

At the time of submission, the medicine was not authorised anywhere in the EU for malignant mesothelioma. 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 23 March 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.

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