EU/3/18/2005: Orphan designation for the treatment of follicular lymphoma

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 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 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 191,000 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 517,400,000 (Eurostat 2018).

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, obinutuzumab, pixantrone and rituximab were authorised for the treatment of follicular lymphoma.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with follicular lymphoma because early results from studies in patients with the condition showed responses to treatment that compared well to those seen with authorised products in other studies. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Tazemetostat blocks the action of a protein called EZH2. Some patients with follicular lymphoma have an altered form of the EZH2 protein, which encourages abnormal growth and development of B cells. By blocking the altered EZH2 protein, tazememostat is expected to lead to the death of the lymphoma cells and thus stop the spread of the lymphoma.

The effects of tazemetostat have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with tazemetostat in patients with follicular lymphoma were ongoing.

At the time of submission, tazemetostat was not authorised anywhere in the EU for follicular lymphoma. Orphan designation of the medicine had been granted in the United States for follicular lymphoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 February 2018 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.