EU/3/15/1470 - orphan designation for treatment of biliary tract cancer
5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl)bacteriochlorin (redaporfin)
OrphanHuman
Overview
This medicine is now known as redaporfin.
On 19 March 2015, orphan designation (EU/3/15/1470) was granted by the European Commission to Luzitin S.A., Portugal, for 5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl) bacteriochlorin for the treatment of 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 which is produced by the liver and released into the intestines after a meal to help digest fats. The cancer is characterised by various clinical 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 which is often diagnosed when the disease has reached a late stage, worsening the prognosis for the patient.
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 77,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 512,900,000 (Eurostat 2015).
At the time of designation, choice of treatment for biliary tract cancer depended mainly on how advanced the disease was. Some patients with early disease could undergo surgery to remove the cancer. Other treatments included chemotherapy (medicines to treat cancer).
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with biliary tract cancer because it works in a different way to existing treatments by specifically targeting the cancer. In addition, the medicine may be used in combination with existing treatments to improve the outcome of patients with the condition. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is a photosensitising agent (a substance that changes when exposed to light). When injected into the patient, the medicine is absorbed into cells throughout the body. When laser light of a specific wavelength is shone at the cancer area, the photosensitising agent is activated and reacts with oxygen in the cells to create toxic molecules containing oxygen called 'reactive oxygen species' which are expected to kill the 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 biliary tract cancer had been started.
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 12 February 2015 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
- 5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl)bacteriochlorin (redaporfin)
- Intended use
- Treatment of biliary tract cancer
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1470
- Date of designation
- Sponsor
Luzitin S.A.
Edificio Bluepharma
Sao Martinho do Bispo
Coimbra 3045-016
Portugal
Tel. +351 239 800 300
Fax +351 239 800 333
E-mail: luzitin@luzitin.pt
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:
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.
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: