EU/3/16/1751 - orphan designation for treatment of peripheral T-cell lymphoma

Fenretinide

Overview

On 14 October 2016, orphan designation (EU/3/16/1751) was granted by the European Commission to Clinipace GmbH, Germany, for fenretinide for the treatment of peripheral 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Please note that this product was withdrawn from the Union Register of orphan medicinal products in January 2022 on request of the Sponsor.

What is peripheral T-cell lymphoma?

Peripheral T-cell lymphoma 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 peripheral T-cell lymphoma there is uncontrolled growth of T lymphocytes (T cells), a type of white blood cell found in the lymphatic system. Peripheral T-cell lymphomas include types that mainly occur in the lymph nodes (primary nodal) and types that occur mainly outside the lymph nodes (primary extranodal).

The symptoms of the disease vary according to the type of lymphoma, but the first sign may be a lump in the neck, under the arm or in the groin area, which is caused by an enlarged lymph node. The lymphoma may also affect other organs in the body such as the bone marrow, liver and the skin.

Peripheral T-cell lymphoma is a long-term debilitating and life-threatening condition because in most cases the disease does not respond well to therapy, usually comes back within one year and is associated with poor overall survival.

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

At the time of designation, peripheral T-cell lymphoma affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 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).

What treatments are available?

At the time of designation, there were no specific treatments for peripheral T-cell lymphoma, but the disease was treated in the same way as the broader class of lymphomas known as non-Hodgkin's lymphomas, for which several medicines were authorised in the EU. The main treatment was chemotherapy (medicines to treat cancer), sometimes in combination with radiotherapy (treatment with radiation).

The sponsor has provided sufficient information to show that fenretinide might be of significant benefit for patients with peripheral T-cell lymphoma because early results suggested it might produce a response in patients who had not responded to previous treatments or whose disease had come back after treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

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 certain toxic ceramides and reactive oxygen species (ROS, toxic molecules containing oxygen) which induce cancer cell death.

What is the stage of development of this medicine?

The effects of fenretinide have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with fenretinide in patients with peripheral T-cell lymphoma were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for peripheral T-cell lymphoma. Orphan designation of fenretinide had been granted in the United States for peripheral T-cell lymphomas and in both the EU and the United States for cutaneous T-cell lymphoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 September 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/3/16/1751: Public summary of positive opinion for orphan designation of fenretinide for the treatment of peripheral T-cell lymphoma

Adopted Reference Number: EMA/622420/2016

Key facts

• Active substance:
  Fenretinide
• Intended use:
  Treatment of peripheral T-cell lymphoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/16/1751
• Date of designation:
  14/10/2016
• Sponsor:

  Clinipace GmbH; Helfmann-Park 10; 65760 Eschborn; Germany; E-mail: [e-mail]