EU/3/15/1579

Follicular lymphoma is a cancer of a type of white blood cell called B lymphocytes or B cells. In follicular lymphoma, the B cells multiply too quickly and live for too long, so there are too many of them in the lymph nodes. The first sign of the disease is usually a lump in the neck, under the arm or in the groin area, caused by an enlarged lymph node. Patients may also have fever, weight loss, tiredness and night sweats.

Follicular lymphoma is usually diagnosed in people aged over 50 years. It is a long-term debilitating and life-threatening disease due to organ damage and the cancer coming back.

At the time of designation, follicular lymphoma affected approximately 2.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 144,000 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 512,900,000 (Eurostat 2015).

At the time of designation, the main treatments for follicular lymphoma available in the EU included chemotherapy (medicines to treat cancer) combined with immunotherapy (medicines that stimulate the body's own immune system to kill the cancer cells). The medicines ibritumomab tiuxetan, idelalisib, interferon alfa-2b and rituximab were specifically authorised for the treatment of follicular lymphoma.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with follicular lymphoma because early studies showed 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 abnormal B cells in patients with follicular lymphoma produce a protein on their surface called CD19.

This medicine is made up of immune cells (called T cells) which are taken from the patient and modified in the laboratory with a virus that carries a gene into the T cells so that they can recognise and attach to CD19. The modified T cells are then given back to the patient, where they are expected to attach to CD19 on the cancer cells and kill them. These T cells are also expected to activate other T cells from the patient to act against the cancer cells.

The type of virus used in this medicine ('retrovirus') is modified in order not to cause disease in humans.

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, a clinical trial with the medicine including patients with follicular lymphoma was ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for follicular 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 8 October 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.