EU/3/16/1720 - orphan designation for treatment of cholangiocarcinoma

Fimaporfin

Overview

On 29 August 2016, orphan designation (EU/3/16/1720) was granted by the European Commission to PCI Biotech AS, Norway, for fimaporfin for the treatment of cholangiocarcinoma.

What is cholangiocarcinoma?

Cholangiocarcinoma is a type of cancer that begins in the bile ducts. These are small tubes through which bile, which is produced by the liver and stored in the gall bladder, enters the intestine where it helps to digest fats. Symptoms usually only occur once the cancer is in its advanced stages, when it may spread to other parts of the body.
Cholangiocarcinoma is a life-threatening condition with a high mortality rate.

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

At the time of designation, cholangiocarcinoma affected less than 1.3 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 67,000 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).

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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).

How is this medicine expected to work?

Fimaporfin is a photosensitising agent (a substance that changes when exposed to light). It is to be used with gemcitabine, a medicine already authorised for the treatment of several types of cancer. When fimaporfin and gemcitabine are injected into a blood vessel, they are distributed throughout the body and reach the bile ducts. When a laser light is targeted at the bile duct cancer, fimaporfin is activated and generates reactive oxygen molecules that modify internal structures of the cancerous bile duct cells. This allows the accompanying gemcitabine to disrupt production of DNA in these cells, thus preventing them from multiplying and slowing down the growth 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, clinical trials with the medicine in patients with cholangiocarcinoma were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for cholangiocarcinoma 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 13 July 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.

EU/3/16/1720 : Public summary of opinion on orphan designation: Fimaporfin for the treatment of cholangiocarcinoma

Adopted

Key facts

• Active substance:
  Fimaporfin
• Intended use:
  Treatment of cholangiocarcinoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1720
• Date of designation:
  29/08/2016
• Sponsor:

  PCI Biotech AS; Ullernchausséen 64; 0379 Oslo; Norway; 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.