EU/3/20/2262 - orphan designation for treatment of biliary tract cancer
fosgemcitabine palabenamide
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2025 on request of the Sponsor.
On 24 March 2020, orphan designation EU/3/20/2262 was granted by the European Commission to Pharma Gateway AB, Sweden, for fosgemcitabine palabenamide (also known as NUC-1031) for the treatment of biliary tract cancer.
Biliary tract cancer affects the bile ducts and gallbladder. These are parts of the digestive system that transport and store bile, a fluid produced by the liver and released into the intestines to help digest fats. The cancer is characterised by features such as abnormal liver function tests, abdominal (belly) pain, yellowish discoloration of the skin and weight loss.
Biliary tract cancer is a long-term debilitating and life-threatening disease due to liver failure and problems caused when the cancer blocks the bile ducts.
At the time of designation, biliary tract cancer affected approximately 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 78,000 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).
*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of biliary tract cancer. Some patients with early disease could undergo surgery to remove the cancer. Other treatments included chemotherapy medicines (medicines to treat cancer), although these were not authorised for biliary tract cancer.
Fosgemcitabine palabenamide is related to another cancer medicine, gemcitabine, that has been widely used in the EU. Like gemcitabine, it is taken up into dividing cells such as cancer cells and incorporated into their genetic material (DNA), interfering with their ability to grow and function normally. This results in death of the affected cells. Because of the differences in its structure, fosgemcitabine palabenamide is able to enter and kill some cancer cells that are resistant to the action of gemcitabine.
The effects of fosgemcitabine palabenamide have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with fosgemcitabine palabenamide in patients with biliary tract cancer were ongoing.
At the time of submission, fosgemcitabine palabenamide was not authorised anywhere in the EU for the treatment of biliary tract cancer. Orphan designation of fosgemcitabine palabenamide had been granted in the United States for the treatment of biliary tract cancer.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 20 February 2020, 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
- fosgemcitabine palabenamide
- Intended use
- Treatment of biliary tract cancer
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/20/2262
- Date of designation
- Sponsor
Pharma Gateway AB
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: