EU/3/16/1756: Orphan designation for the treatment of cutaneous T-cell lymphoma

Cutaneous T-cell lymphoma (CTCL) is a cancer of the T lymphocytes (T cells), a type of white blood cell. 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 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 134,000 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 mogamulizumab might be of significant benefit for patients with CTCL because early studies show that patients whose disease had come back after 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.

This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to receptors called C-C chemokine receptor 4 (CCR4), which are found in high amounts on the surface of CTCL cells. By attaching to CCR4, this medicine is expected to activate cells of the immune system (the body's natural defences), which then kill the cancer cells.

The effects of mogamulizumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with mogamulizumab in patients with CTCL were ongoing.

At the time of submission, mogamulizumab was authorised in Japan for CTCL.

At the time of submission, mogamulizumab was not authorised anywhere in the EU for CTCL. Orphan designation of mogamulizumab had been granted in the US and Japan for CTCL.

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.

• 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.