EU/3/18/2121 - orphan designation for treatment of biliary tract cancer

sodium 2-hydroxylinoleate

Overview

On 14 December 2018, orphan designation (EU/3/18/2121) was granted by the European Commission to Ability Pharmaceuticals SL, Spain, for sodium 2-hydroxylinoleate (also known as ABTL0812) for the treatment of biliary tract cancer.

What is biliary tract cancer?

Biliary tract cancer is cancer of 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 after a meal to help digest fats. The cancer is characterised by various features such as abnormal liver function tests, pain in the belly, 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.

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

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).

---

*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 517,400,000 (Eurostat 2018).

What treatments are available?

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.

How is this medicine expected to work?

This medicine is expected to work by reducing the activity of several proteins in the ‘Akt/mTOR signalling pathway’. This is a mechanism within cells which is important in regulating their growth and survival. In many cancers, including biliary tract cancer, this pathway is overactive, allowing the cancer cells to grow uncontrollably. By reducing the activity of this pathway, the medicine is expected to slow down the progression of the cancer.

What is the stage of development of this medicine?

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 biliary tract cancer were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for biliary tract 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 8 November 2018 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/3/18/2121: Public summary of opinion on orphan designation: Sodium 2-hydroxylinoleate for the treatment of biliary tract cancer

Adopted Reference Number: EMA/833241/2018

Key facts

• Active substance:
  sodium 2-hydroxylinoleate
• Intended use:
  Treatment of biliary tract cancer
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2121
• Date of designation:
  14/12/2018
• Sponsor:

  Ability Pharmaceuticals SL; Edifici Eureka; Campus de la UAB; 08193 Bellaterra; Spain; Tel. [phone]; E-mail: [e-mail]

Review of designation

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