EU/3/16/1630 - orphan designation for treatment of cutaneous T-cell lymphoma
Fenretinide
OrphanHuman
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Overview
On 21 March 2016, orphan designation (EU/3/16/1630) was granted by the European Commission to Clinipace GmbH, Germany, for fenretinide for the treatment of cutaneous T-cell lymphoma.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in January 2022 on request of the Sponsor.
Cutaneous T-cell lymphoma (CTCL) is a cancer of the lymphatic system, a network of vessels that transport fluid from tissues through the lymph nodes and into the bloodstream.
In CTCL there is uncontrolled growth of the T lymphocytes (T cells), a type of white blood cell found in the lymphatic system. The cancerous T cells appear in the skin, causing lesions (rashes, plaques and tumours) which can be itchy and painful.
CTCL usually happens in people aged between 40 and 60 years. In many cases, patients survive a long time with the disease; however, in some cases the disease can be serious and life threatening because it can develop into more aggressive forms of cancer and may have a large impact on quality of life, particularly because the skin lesions can cause disfigurement.
At the time of designation, CTCL affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 102,000 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 513,700,000 (Eurostat 2016).
At the time of designation, several products were authorised for the treatment of CTCL within the EU. Treatments for CTCL can be divided into topical (applied to the skin) and systemic (affecting the whole body):
- topical treatments included topical corticosteroids, the topical cancer medicine carmustine and ultraviolet light;
- systemic treatments included cytotoxic medicines (medicines that kill cells that are dividing, such as cancer cells) and interferon alfa (a medicine that helps the immune system to fight against the cancer cells).
The sponsor has provided sufficient information to show that fenretinide might be of significant benefit for patients with CTCL because early studies show that patients whose disease had come back after previous treatment or did not respond to previous treatment responded to treatment with this medicine. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The exact mechanism of action of fenretinide is not fully understood but it is thought to cause death of cancer cells through different mechanisms such as the production of ceramide and reactive oxygen species (ROS, toxic molecules containing oxygen) which induce cancer cell death.
The effects of fenretinide 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 CTCL were ongoing.
At the time of submission, fenretinide was not authorised anywhere in the EU for CTCL. Orphan designation of the medicine had been granted in the United States for cutaneous T-cell lymphoma and for peripheral T-cell lymphoma.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 February 2016 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
- Fenretinide
- Intended use
- Treatment of cutaneous T-cell lymphoma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/16/1630
- Date of designation
- Sponsor
Clinipace GmbH
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: