EU/3/19/2243 - orphan designation for treatment of pancreatic cancer
motixafortide
OrphanHuman
Overview
On 9 January 2020, orphan designation EU/3/19/2243 was granted by the European Commission to Granzer Regulatory Consulting & Services, Germany, for motixafortide 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, most patients are diagnosed when the cancer has spread nearby or to other parts of the body.
Pancreatic cancer is a severe and life-threatening disease that can shorten life expectancy.
At the time of designation, pancreatic cancer affected approximately 1.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 93,000 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. This represents a population of 518,400,000 (Eurostat 2019).
At the time of designation, several medicines were authorised in the EU for treating pancreatic cancer. The choice of treatment depended on several factors, including how far the disease had advanced. 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 advanced pancreatic cancer. In early studies in patients with advanced pancreatic cancer, the tumour reduced in size when the medicine was used in combination with another cancer medicine called a ‘checkpoint inhibitor.’
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine blocks CXC chemokine receptor 4 (CXCR-4), which plays a role in preventing cells of the immune system (the body’s natural defences) from attacking cancer cells and in promoting the spread of the cancer. By blocking CXCR4, the medicine stops the cancer switching off the immune cells, thereby increasing the immune system’s ability to kill the cancer cells, and may slow down the spread of the cancer. Motixafortide also mobilises cancer cells to make them more prominent targets of other cancer medicines such as pembrolizumab.
The effects of motixafortide have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with motixafortide in patients with pancreatic cancer were ongoing.
At the time of submission, motixafortide was not authorised anywhere in the EU for the treatment of pancreatic cancer. Orphan designation had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 5 December 2019, 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.
Key facts
- Active substance
- motixafortide
- Intended use
- Treatment of pancreatic cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/19/2243
- Date of designation
- Sponsor
Granzer Regulatory Consulting & Services
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: