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On 16 October 2017, orphan designation (EU/3/17/1917) was granted by the European Commission to Sagetis Biotech, S.L., Spain, for adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters (also known as SAG-101), for the treatment of pancreatic cancer.
Pancreatic cancer is a cancer of the pancreas, a small organ that lies behind the stomach. The pancreas has two functions: to produce a fluid that helps with the digestion of food, and to produce hormones such as insulin. Due to the absence of symptoms in the early stages of pancreatic cancer, the majority of patients are diagnosed when the cancer has spread nearby or to other parts of the body.
Pancreatic cancer is a very severe and life-threatening disease that is associated with shortened life expectancy.
At the time of designation, pancreatic cancer affected approximately 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 124,000 people1, 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).
1Disclaimer: 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 515,700,000 (Eurostat 2017).
At the time of designation, several medicines were authorised in the EU for treating pancreatic cancer. Treatments included surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with pancreatic cancer. Laboratory studies showed that the tumour shrank when the medicine was used in combination with authorised chemotherapy.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is an advanced therapy that belongs to the group called 'gene therapy products'. These are medicines that work by delivering genes into the body.
The medicine is made up of an 'oncolytic' virus, a virus that has been genetically modified so that it is able to target, multiply in and destroy cancer cells while sparing normal cells. The virus is coated with a substance that protects it from the immune system (the body's natural defences) and allows it to be drawn to the cancer. When inside a cancer cell, the virus is expected to take over the cell's replication apparatus and use it to make more copies of itself. This is expected to kill the cell, leaving the virus to spread to neighbouring cancer cells.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with pancreatic cancer had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for pancreatic cancer 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 7 September 2017 recommending the granting of this designation.
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.
Sagetis Biotech, S.L.
Via Augusta, 394
08017 Barcelona
Spain
Tel. +34 933097169
E-mail: xrivero@sagetis-biotech.com
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: