EU/3/17/1942 - orphan designation for treatment of peripheral T-cell lymphoma

N-(2-aminophenyl)-4-(1-[(1,3-dimethyl-1H-pyrazol-4-yl)methyl]piperidin)benzamide

Overview

On 12 October 2017, orphan designation (EU/3/17/1942) was granted by the European Commission to Celleron Therapeutics Limited, United Kingdom, for N-(2-aminophenyl)-4-(1-[(1,3-dimethyl-1H-pyrazol-4-yl)methyl]piperidin)benzamide (also known as CXD101) for the treatment of peripheral T-cell lymphoma.

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 early death.

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 52,000 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).

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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 515,700,000 (Eurostat 2017).

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 this medicine might be of benefit for patients with peripheral T-cell lymphoma because early results suggested that it might produce a response in patients whose disease had not responded to previous treatments or 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?

This medicine is an 'HDAC inhibitor'. This means that it blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes 'on' and 'off' within cells. By blocking HDACs, the medicine is expected to 'switch on' the genes that suppress the division and growth of the tumour cells in peripheral T-cell lymphoma. This is expected to lead to a reduction in the growth and division of the cancer cells.

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 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 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 5 October 2017 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/3/17/1942: Public summary of opinion on orphan designation: N-(2-aminophenyl)-4-(1-[(1,3-dimethyl-1H-pyrazol-4-yl)methyl]piperidin)benzamide for the treatment of peripheral T-cell lymphoma

AdoptedReference Number: EMA/694349/2017

Key facts

• Active substance:
  N-(2-aminophenyl)-4-(1-[(1,3-dimethyl-1H-pyrazol-4-yl)methyl]piperidin)benzamide
• Intended use:
  Treatment of peripheral T-cell lymphoma
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1942
• Date of designation:
  12/10/2017
• Sponsor:

  Celleron Therapeutics Limited; Magdalen Centre; Robert Robinson Avenue; Oxford Science Park; Oxford OX4 4GA; United Kingdom; 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.